2015 Annual Report - Center for International Blood and Marrow … · 2016-02-01 · Suite C5500 ....

CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program®/Be The Match® and Medical College of Wisconsin

Sharing Knowledge. Sharing Hope.

2015 Annual Report

2015 Annual Report

January – December

Milwaukee Campus Medical College of Wisconsin 9200 W Wisconsin Ave, Suite C5500 Milwaukee, WI 53226 (414) 805-0700

Minneapolis Campus National Marrow Donor Program/ Be The Match 500 N 5th St Minneapolis, MN 55401 (612) 627-5800

cibmtr.org

CIBMTR 2015 Annual Report TABLE OF CONTENTS

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TABLE OF CONTENTS

1.0 WHO WE ARE ........................................................................................................................................ 1

1.1 Mission .............................................................................................................................................. 1

1.2 Value to the HCT Community ............................................................................................................ 1

1.3 Organizational Structure ................................................................................................................... 1

1.3.1 Scientific Working Committees .................................................................................................. 6

2.0 WHAT WE DO ........................................................................................................................................ 8

2.1 Clinical Outcomes Research Program ............................................................................................. 11

2.1.1 Scientific Working Committees ................................................................................................ 11

2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives ..................................... 15

2.2 Immunobiology Research Program ................................................................................................. 16

2.3 Clinical Trials Support Program ....................................................................................................... 18

2.3.1 Blood and Marrow Transplant Clinical Trials Network ............................................................ 18

2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation ............................ 20

2.4 Health Services Research Program.................................................................................................. 21

2.5 Bioinformatics Research Program ................................................................................................... 23

2.6 Statistical Methodology Research Program .................................................................................... 24

2.7 Stem Cell Therapeutic Outcomes Database (SCTOD) ..................................................................... 25

2.8 Corporate Program .......................................................................................................................... 27

3.0 HOW WE SHARE KNOWLEDGE ........................................................................................................... 28

3.1 Information Request Service ........................................................................................................... 33

3.2 Internet Presence ............................................................................................................................ 34

3.2.1 CIBMTR Public Website ............................................................................................................ 34

3.2.2 CIBMTR Collaborative Site ........................................................................................................ 35

3.2.3 CIBMTR Portal Site .................................................................................................................... 36

3.2.4 Be The Match Public Website................................................................................................... 37

3.2.5 Be The Match Clinical Website ................................................................................................. 37

3.2.6 HRSA Blood Cell Transplant Website........................................................................................ 37

3.2.7 Other Applications and Data Exchange Standards ................................................................... 38

3.3 BMT Tandem Meetings ................................................................................................................... 39

3.4 Data Management Training ............................................................................................................ 40

4.0 HOW WE COLLECT AND MANAGE DATA ............................................................................................ 41

4.1 Research Data Life Cycle ................................................................................................................. 41

4.2 Collecting and Storing Data ............................................................................................................. 42

4.2.1 FormsNet .................................................................................................................................. 42

4.2.2 Research Database ................................................................................................................... 42

4.3 Ensuring Data Quality ...................................................................................................................... 42


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4.3.1 Continuous Process Improvement ........................................................................................... 42

4.3.2 Verification and Validation ....................................................................................................... 43

4.3.3 On-Site Data Audit Program ..................................................................................................... 44

4.4 Protecting Patients and Data .......................................................................................................... 45

4.4.1 Human Subjects / HIPAA Compliance ...................................................................................... 45

4.4.2 Information Security and Data Privacy ..................................................................................... 45

5.0 WHAT WE WILL DO NEXT.................................................................................................................... 46

2015 KEY ACCOMPLISHMENTS ................................................................................................................. 49

APPENDIX A: TRANSPLANT CENTERS ........................................................................................................ 53

Appendix A1: US Centers....................................................................................................................... 54

Appendix A2: International Centers ...................................................................................................... 65

APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP ..................... 74

Appendix B1: Organizational Structure – Milwaukee Campus ............................................................. 75

Appendix B2: Organizational Structure – Minneapolis Campus ........................................................... 76

Appendix B3: Coordinating Center Leadership ..................................................................................... 78

APPENDIX C: COMMITTEE MEMBERSHIP ................................................................................................. 87

Appendix C1: Advisory Committee Membership .................................................................................. 87

Appendix C2: Executive Committee Membership ................................................................................ 90

Appendix C3: Consumer Advocacy Committee Membership ............................................................... 92

Appendix C4: Nominating Committee Membership ............................................................................. 93

Appendix C5: Scientific Working Committee Leadership ..................................................................... 94

Appendix C6: Immunobiology Steering Committee Membership ........................................................ 98

Appendix C7: Clinical Trials Advisory Committee Membership ............................................................ 99

APPENDIX D: PUBLICATIONS ................................................................................................................... 100

Appendix D1: Scientific Working Committee Publications ................................................................. 100

Appendix D2: BMT CTN Publications .................................................................................................. 112

Appendix D3: Health Services Research Program Publications .......................................................... 115

Appendix D4: Bioinformatics Research Program Publications ........................................................... 116

Appendix D5: Statistical Methodology Research Program Publications ............................................ 119

Appendix D6: Coordinating Center Publications ................................................................................. 120

APPENDIX E: PRESENTATIONS ................................................................................................................ 124

APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ..................................................... 132

APPENDIX G: CLINICAL TRIALS ................................................................................................................ 135

Appendix G1: BMT CTN Clinical Trials Open for Enrollment ............................................................... 135

Appendix G2: RCI BMT Clinical Trials .................................................................................................. 137

APPENDIX H: FORMS SUBMISSION PROCESS ......................................................................................... 140

APPENDIX I: WEBSITES ............................................................................................................................ 141

APPENDIX J: GLOSSARY ........................................................................................................................... 142


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FIGURES AND TABLES Figure 1.1. Scientific Organizational Structure ........................................................................................... 2

Figure 1.2. Functional Organizational Structure with Scientific Oversight ................................................. 3

Table 1.3. Committee Structure ................................................................................................................. 4

Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source .......... 8

Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease .................. 9

Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR ......................... 10

Table 2.4. 2015 Working Committee Studies ........................................................................................... 12

Figure 2.5. 2015 Publications by Program ................................................................................................ 13

Figure 2.6. Working Committee Study Proposal Review Process............................................................. 14

Figure 3.1. How to Access CIBMTR Knowledge ........................................................................................ 28

Table 3.2. How to Access CIBMTR Information ........................................................................................ 29

Table 3.3 How to Access CIBMTR Data ..................................................................................................... 29

Table 3.4 How to Access CIBMTR Tools .................................................................................................... 30

Table 3.5 How to Access CIBMTR Biospecimens ...................................................................................... 30

Table 3.6. Standard Reports Published by the CIBMTR ............................................................................ 31

Table 3.7. Data Requests Addressed by the CIBMTR in 2015................................................................... 33

Figure 4.1. Research Data Life Cycle ......................................................................................................... 41

Figure 4.2. Audit Process .......................................................................................................................... 44

Figure 5.1. Plans for 2016 ......................................................................................................................... 46

Table 5.2. Plans to Enhance Data .............................................................................................................. 47

Table 5.3. Plans to Expand Knowledge Sharing ........................................................................................ 47

Table 5.4. Plans to Increase Impact .......................................................................................................... 48

Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR ......................................... 53

CIBMTR 2015 Annual Report 1.0 WHO WE ARE

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1.0 WHO WE ARE

The CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW).

1.1 MISSION The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and enrich quality of life for patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database.

CIBMTR Programs Clinical Outcomes Research

Immunobiology Research

Clinical Trials Support

• Blood and Marrow Transplant Clinical Trials Network (BMT CTN)

• Resource for Clinical Investigations in Blood and Marrow Transplantation (RCI BMT)

Health Services Research

Bioinformatics Research

Statistical Methodology Research

1.2 VALUE TO THE HCT COMMUNITY The CIBMTR has been collecting HCT outcomes data worldwide for >40 years, resulting in a Research Database with information on >425,000 patients. These data are available to investigators with interest in HCT and treatments for cancer and other life-threatening diseases. The CIBMTR has become a respected leader in HCT research by providing a unique resource of information and expertise to the medical and scientific communities.

1.3 ORGANIZATIONAL STRUCTURE The CIBMTR (Figures 1.1 and 1.2) represents a large network of approximately 400 participating transplant centers (Appendix A) that submit transplant-related data for patients. Centers submit data at two levels: a Transplant Essential Data (TED) level, which captures basic data, and a Comprehensive Report Form (CRF) level, which captures more detail. The CIBMTR Coordinating Center, staffed by almost 200 employees (Appendix B), provides data acquisition, management, and statistical support for analyses of these data.

The Chief Scientific Director is responsible for all administrative and scientific operations. The Associate and Senior Scientific Directors assist in overseeing operational aspects of the Coordinating Center. CIBMTR committees (Table 1.3) provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of the scientific and medical communities.


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Figure 1.1. Scientific Organizational Structure

Joint Affiliation CommitteeExecutive Director

J Chell, MD

National Marrow Donor Program/Be The MatchTransplant Centers

CIBMTR Assembly

Advisory Committee

Executive Committee

Medical College of Wisconsin

Senior Research AdvisorD Weisdorf, MD

Senior Scientific Director for Research Operations

M Eapen, MBBS, MS

Senior Scientific Director for SCTOD

JD Rizzo, MD, MS

Associate Scientific Director for CIBMTR

MinneapolisD Confer, MD

Chief Scientific DirectorM Horowitz, MD, MS

Scientific Working CommitteesSteering Committees

Consumer Advocacy Committee

Chief Statistical Director

MJ Zhang, PhD

Statistical Methodology

Research ProgramMJ Zhang, PhD

Clinical Outcomes Research Program

M Eapen, MBBS, MS

Health Services Research Program

L Burns, MD

Immunobiology Research Program

S Lee, MD, MPH

Clinical Trials Support Program

D Confer, MDL Burns, MD

M Pasquini, MD, MSB Shaw, MD, PhD

Blood and Marrow Transplant Clinical Trials Network

(BMT CTN)M Pasquini, MD, MS

Resource for Clinical Investigations in Blood and Marrow Transplantation

(RCI BMT)D Confer, MDL Burns, MD

B Shaw, MD, PhD

Advisory role Senior Scientific Director for Data OperationsB Shaw, MD, PhD

Advisory role

Bioinformatics Research Program

M. Maiers, MS


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Figure 1.2. Functional Organizational Structure with Scientific Oversight

Research & AdministrationM Horowitz & D Confer

CIBMTR Administration

P Steinert & R King

Finance, Grants & Contracts

MCW & NMDP/Be The Match

Faculty

M Horowitz & D Confer

InformationTechnology

E Bergman & M PrestegaardMKE & MPLS

(M Eapen)(D Rizzo)(B Shaw)

Corporate

S FisherMKE

(M Eapen)

Advancement

S Fisher &T HousemanMKE & MPLS

(M Horowitz)

BMT CTN

A Foley MPLS

(M Pasquini)

Data Operations

J Brunner & M Matlack

MKE & MPLS

(B Shaw)

Clinical Studies Support

R Drexler

MPLS

(D Confer)(L Burns)(B Shaw)

Quality Assurance

TBDMKE

(M Horowitz)

Human Research Protection Program

R KingMPLS

(**B Lindberg)

Auditing & Monitoring

D Christianson

MPLS

(B Shaw)

Statistics & Observational

Research

W Perez &S Spellman

MKE & MPLS

(M Eapen)

Immunobiology Research

S SpellmanMPLS

(S Lee)

Statistics & Clinical

Outcomes

W Perez & S Spellman

MKE & MPLS

(MJ Zhang)(M Eapen)

SurveyResearch

Group

D MattilaMPLS

(D Confer)

RCI BMT

R DrexlerMPLS

(D Confer)(L Burns)(B Shaw)

Database Administrator

& Data Architect

B Liu &

TBDMKE & MPLS

Data Solutions

R RennerMPLS

Project Management& Analysis

T Moerke& K Gee

MKE & MPLS

Applications

T Hongyu& E Chan

MKE & MPLS

Recipient Data

Management

S Meiers & K Gardner

MKE & MPLS

(B Shaw)

Donor Data Management

A HauckMPLS

(D Confer)

Data Entry & Imaging

B LevesqueMPLS

(B Shaw)

Training

M MatlackMPLS

(B Shaw)

Data Management

Special Projects

J Brunner & M Matlack

MKE & MPLS

(B Shaw)

MDs

PhDs

Data Support

M MatlackMPLS

(B Shaw)

TechnicalServices

T MoerkeMKE

Health Services

Research*

E DenzenMPLS

(L Burns)

Key: MKE = Milwaukee, MPS = Minneapolis, ( ) indicates scientific oversight, *staffed through NMDP/Be The Match Patient and Health Professional Services, ** institutional official oversight

Meetings

T HousemanMPLS

(M Horowitz)

Communications

S FisherMKE

(M Horowitz)

Bioinfomatics

M MaiersMPLS

Business Office

C Gonzalez &P Vespalec

MKE


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Table 1.3. Committee Structure

Committee Function Meetings Roster

Joint Affiliation Board • Reviews and approves the CIBMTR budget and research plan • Amends the terms of the affiliation agreement, as necessary • Reviews and approves data access and confidentiality policies

• Annually

Assembly • Includes representatives from each transplant center that submits CRF-level data

• Elects members of the Advisory, Nominating, and Clinical Trials Advisory Committees

• Annually during the BMT Tandem Meetings

Advisory Committee • Oversees CIBMTR policies and scientific agenda • Partners with the Working Committees to prioritize scientific

studies

• In person annually at the BMT Tandem Meetings

• By teleconference quarterly and as needed

Appendix C1

Executive Committee (subcommittee of Advisory Committee)

• Provides scientific and policy advice to the Chief Scientific Director and Coordinating Center

• Reviews audit results and makes recommendations for improvement

• Four times annually by teleconference

Appendix C2

Consumer Advocacy Committee (subcommittee of Advisory Committee)

• Provides patient and donor perspectives during the development of the CIBMTR research agenda

• Communicates CIBMTR research results and data to the non-medical community


• By teleconference periodically

Appendix C3

Nominating Committee

• Prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees

• Makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments

• At least once annually each Fall by teleconference

Appendix C4


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Committee Function Meetings Roster

Scientific Working Committees (Section 1.3.1)

• Design and conduct relevant studies using CIBMTR data, statistical resources, networks, and / or centers

• Set priorities for clinical outcomes studies • Assess and revise CIBMTR data collection forms, as needed • Plan and conduct workshops at CIBMTR meetings


• Leadership - by teleconference every 4-8 weeks

Leadership - Appendix C5

Immunobiology Steering Committee / NMDP/Be The Match Histocompatibility Advisory Group

• Reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies

• In person twice annually, in summer and at the BMT Tandem Meetings

Appendix C6

Clinical Trials Advisory Committee

• Assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT


• By teleconference as needed

Appendix C7


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1.3.1 Scientific Working Committees

To ensure broad input into the research process and efficient use of resources, the CIBMTR facilitates 15 Scientific Working Committees focused on specific research areas.

Scientific Working Committees Acute Leukemia

Autoimmune Diseases and Cellular Therapies

Chronic Leukemia

Donor Health and Safety

Graft Sources and Manipulation

Graft-versus-Host Disease

Health Services and International Studies

Immunobiology

Infection and Immune Reconstitution

Late Effects and Quality of Life

Lymphoma

Pediatric Cancer

Plasma Cell Disorders and Adult Solid Tumors

Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders

Regimen-Related Toxicity and Supportive Care

Total Working Committee membership exceeds 2,300 researchers. Membership is open to any researcher willing to take an active role in developing and conducting studies that use CIBMTR data and / or resources. While most of these individuals are HCT clinicians, statisticians and basic scientists also participate. PhD-level statistical faculty and Master’s-level statisticians from the CIBMTR Coordinating Center provide their unique expertise in data analysis. Basic scientists investigating human leukocyte antigen (HLA), immunogenetics,

pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective research areas. The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities.

Working Committee Leadership Chairs (usually 3-4)

MD Scientific Director

PhD Statistical Director

MS-level Statistician

Working Committee leadership is listed in Appendix C5.

1.3.1.1 Working Committee Chairs

Working Committee Chairs are appointed by the Advisory Committee to non-renewable five-year terms. Appointments are made each fall, with terms commencing on March 1 of the following year. Terms are staggered to facilitate succession and maintain continuity. Individuals may serve as Chair more than once but not consecutively for the same committee. The Nominating Committee polls the CIBMTR membership for potential Chair candidates for open Chair positions. Current Chairs participate in the nomination process for replacement positions and give special consideration to promising junior investigators, thus promoting ongoing leadership for the work of the CIBMTR.

Working Committee Chairs provide subject matter expertise in autologous and allogeneic transplantation as well as understanding of CIBMTR organization and procedures. They must be members of CIBMTR centers that submit CRFs and that are compliant with Continuous Process Improvement (CPI) standards for data submission, unless an exception is granted by the Advisory Committee. Chairs are occasionally selected from outside these guidelines for their specific


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scientific expertise, for example, a scientist who directs a histocompatibility laboratory, apheresis center, or donor registry, who is committed to the CIBMTR and to the field of HCT.

Chairs monitor and facilitate the progress of studies in their Working Committee’s portfolio. They communicate with Principal Investigators to address barriers and / or delays in study completion and participate in weekly CIBMTR Coordinating Center study critiques when studies in their portfolios are being discussed. In addition to chairing annual Working Committee meetings, Chairs meet by teleconference every four to six weeks with their committee’s Scientific Director and biostatisticians to review the progress of study proposals and ongoing studies. Chairs lead the annual Working Committee meeting and, using input from that meeting, prioritize studies and set the research agenda for the following year.

CIBMTR 2015 Annual Report 2.0 WHAT WE DO

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2.0 WHAT WE DO

The CIBMTR collects data for approximately 21,000 new transplant patients annually as well as a continually increasing volume of follow-up data on previously reported recipients and donors. Submission of outcomes data is mandatory for allogeneic transplants in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous transplants are reported.

The CIBMTR Research Database contains information on >425,000 patients. The distribution of patients in the database is displayed by graft type in Figure and Table 2.1 and by disease in Figure and Table 2.2.Figure 2.3 shows the continued growth in the number of patients registered with the CIBMTR.

Publications

There were 76 CIBMTR publications in 2015. As of December 31, an additional 17 manuscripts were submitted and are under review. A complete list of publications is provided in Appendix D.

Presentations

In 2015, CIBMTR study investigators presented 65 abstracts (41 oral and 24 poster) at national and international conferences, a 50% increase from last year. Presentations included 25 (17 oral and 8 poster) at the American Society for Hematology (ASH) Annual Meeting and 17 (13 oral and 4 poster) at the BMT Tandem Meetings. A complete list of presentations is provided in Appendix E.

Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source

Graft Source Allogeneic Autologous

TED CRF TED CRF Bone Marrow 49,509 58,935 9,885 5,887 Peripheral Blood 73,780 33,848 148,822 34,858 Cord Blood 5,408 8,721 136 8 TOTAL 128,697 101,504 158,843 40,753


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Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease

Disease Allogeneic Autologous TED CRF TED CRF

Acute myelogenous leukemia 40,448 28,231 5,721 2,379 Acute lymphoblastic leukemia 21,096 16,616 1,089 469 Chronic myelogenous leukemia 13,914 15,163 414 282 MDS / myeloproliferative syndromes 12,805 10,635 182 86 Lymphoma 15,793 8,009 64,110 13,514 Plasma cell disorders 4,141 2,169 57,615 11,594 Other malignant diseases1 6,039 3,794 28,917 12,247 Severe aplastic anemia 5,981 6,753 14 8 Inherited erythrocyte disorders2 6,085 7,133 781 174 Immune deficiency disorders 2,395 3,001 0 0 TOTAL 128,697 101,504 158,843 40,753 1. Includes other leukemia (allogeneic, n=8,279; autologous, n=910), solid tumors

(allogeneic, n=1,554; autologous, n=40,254) 2. Includes inherited erythrocyte disorders (allogeneic, n=8,602; autologous, n=9),

inherited platelet disorder (allogeneic, n=186; autologous, n=4), inborn errors of metabolism (allogeneic, n=2,499; autologous, n=0), histiocytic disorders (allogeneic, n=1,413; autologous, n=16), autoimmune disease (allogeneic, n=125; autologous, n=555), other disorders (allogeneic, n=393; autologous, n=371)


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Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR


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2.1 CLINICAL OUTCOMES RESEARCH PROGRAMClinical outcomes research using the CIBMTR Research Database is a core activity of the organization. These studies address a wide range of issues, focusing on questions that are difficult or impossible to address in single-center studies or randomized trials because diseases treated with HCT are uncommon, single centers treat few patients with a given disorder, and not all important questions are amenable to a randomized research design.

2.1.1 Scientific Working Committees

Program Activities

The 15 Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research. There are currently 176 studies in progress (Table 2.4). These studies and those recently published are detailed in the 2015 Report on Working Committee Studies. For the 2015 BMT Tandem Meetings, the Working Committees reviewed 157 new study proposals, 73 of which were presented and 35 of which were approved. The prioritization and selection process ensures that the most important issues can be addressed in a timely manner.

Publications

In 2015, Working Committee study investigators published 38 manuscripts in peer-reviewed journals, about 50% of the total number of CIBMTR publications this year (Figure 2.5). As of December 31, an additional 15 manuscripts were submitted for publication and are under review. A complete list of Working Committee publications is provided in Appendix D1.

Key Working Committee Publications this Year

Chen Y-B et al. Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous HCT. Biology of Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. Epub 2015 Feb 13. PMC4426014.

Sorror ML et al. Prospective validation of the predictive power of the HCT comorbidity index: a CIBMTR® study. Biology of Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479-1487. Epub 2015 Apr 7. PMC4512746.

Orchard PJ et al. HCT for infantile osteopetrosis. Blood. 2015 Jul 9; 126(2):270-276. Epub 2015 May 26. PMC4497967.

Ciurea SO et al. Haploidentical transplant with post-transplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. Epub 2015 Jun 30.

Kollman C et al. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood. Epub 2015 Nov 2.

Presentations

In 2015, Working Committee study investigators presented 38 abstracts (26 oral and 12 poster), including 19 (11 oral and 8 poster) at the ASH Annual Meeting and 10 (9 oral and 1 poster) at the BMT Tandem Meetings. A complete list of CIBMTR presentations is provided in Appendix E.


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Table 2.4. 2015 Working Committee Studies

Working Committee Studies in Progress Publications Presentations

Acute Leukemia 13 3 4

Autoimmune Diseases and Cellular Therapies 6 0 0

Chronic Leukemia 12 0 3

Donor Health and Safety 11 2 6

Graft Sources and Manipulation 5 3 0

Graft-versus-Host Disease 10 2 2

Health Services and International Studies 12 2 4

Immunobiology 43 6 5

Infection and Immune Reconstitution 9 1 0

Late Effects and Quality of Life 11 2 2

Lymphoma 6 6 7

Pediatric Cancer 4 2 0

Plasma Cell Disorders and Adult Solid Tumors 12 2 2

Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders

10 3 0

Regimen-Related Toxicity and Supportive Care 12 6 3

TOTAL 176 38* 38

*Two studies are joint studies between two committees.


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Funding

Support for the Working Committees is primarily provided by the National Institutes of Health (NIH) grant # U24CA076518 from the National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute for Allergy and Infectious Disease (NIAID).

How to Get Involved

Working Committees are collaborative in nature, and all interested individuals are encouraged to participate. Please feel free to attend annual in-person meetings of the Working Committees at the BMT Tandem Meetings in February. Additionally, anyone willing to follow the study development and management process (Appendix F) is eligible to propose a study to the Working Committees (Figure 2.6).

Successful Working Committee Study Proposals are

Feasible. Utilize data available in the CIBMTR Research Database.

Unique. Fill a gap not addressed by current studies or publications.

Important. Impact the field by improving transplant procedures or results.

See the CIBMTR How to Propose a Study webpage for additional guidelines and advice as well as the Study Proposal Outline.

Figure 2.5. 2015 Publications by Program

Clinical Outcomes -

Working Committees

(38)

BMT CTN (8)

Coordinating Center

(14)

Bio- informatics

(9)

Statistical Methodology

(5)

Health Services (4)

Other (9)

http://www.cibmtr.org/Studies/Observational/ProposeStudy/pages/index.aspx


http://www.cibmtr.org/Studies/Observational/ProposeStudy/ProposalOutline/Pages/index.aspx

http://www.cibmtr.org/Studies/Observational/ProposeStudy/ProposalOutline/Pages/index.aspx


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Figure 2.6. Working Committee Study Proposal Review Process

Submission

• By November 15, study investigator submits proposal to the CIBMTR Coordinating Center for consideration at the next BMT Tandem Meetings.

Initial Review

• Working Committee Leaderships reviews for feasibility with CIBMTR data, potential conflict with active studies, scientific merit, and ability to complete the study in a timely fashion. Researchers with similar concepts may be advised to combine their proposals.

Preliminary Assessment

• If Working Committee Leadership clears the proposal to move forward, the MS-level Statistician contacts the study investigator and prepares a table of characteristics of patient data based on the population defined in the proposal.

Presentation

• Study investigator presents the proposal at the Working Committee meeting at the February BMT Tandem meetings.

Voting

• Working Committee members vote for each proposal, assigning a scientific impact score to each.

Final Approval

• Working Committee Leadership utilizes member feedback in determining which proposals to pursue. Advisory Committee approves the CIBMTR research agenda.

Notification

• Working Committee Leadership contacts study investigator to notify of study approval / rejection by the end of April.


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2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives

With the consensus and support of its Advisory Committee, the CIBMTR is committed to collecting data on cellular therapies and non-transplanted patient populations. The CIBMTR amended its registration of cases to accommodate data collection for cellular and other therapies that may not involve transplantation. Additionally, the CIBMTR is working collaboratively with the Primary Immune Deficiency Disease Consortium to add transplant outcomes data to data on non-transplant therapy collected by the Consortium.

Cellular Therapies

In 2015, the CIBMTR initiated a Cellular Therapies Task Force charged with determining appropriate content and structure for cellular therapies data collection. The CIBMTR also hosted a Cellular Therapies Forum in October 2015 to discuss how to further develop and provide recommendations for a cellular therapy registry. Participants included physicians, scientists, manufacturers, and government representatives.

Cellular Therapies Forum Discussion Topics

Cell therapy for treatment of viral infection

CAR-T cells for malignancies

Manufacturing models

Regulatory framework and long-term outcomes reporting

NCI Cancer Immunotherapy Trials Network

Proposed CIBMTR Cellular Therapy Registry

Myelodysplastic Syndrome

The CIBMTR collects data on patients receiving non-HCT therapy for myelodysplastic syndrome (MDS) to compare with an ongoing study of HCT outcomes. By combining its resources with those of the BMT CTN (Section 2.3.1), the CIBMTR is able to do this in a cost-effective manner. Through November 2015, 34 centers have been activated and 141 patients enrolled on this non-HCT therapy study.

The HCT outcomes study was launched in 2010 because many elderly patients with MDS were denied access to HCT therapy in the US due to lack of Medicare insurance coverage by the Centers for Medicare and Medicaid Services (CMS). To help secure Medicare coverage for these patients, the CIBMTR, NMDP/Be The Match, American Society for Blood and Marrow Transplantation (ASBMT), and other organizations partnered with CMS to develop a Coverage with Evidence Development (CED) study, using data in the CIBMTR Research Database that are collected to fulfill SCTOD requirements. The CED approach allows CMS to provide coverage for procedures and to advocate for clinical studies that inform policy decisions. The CMS-approved study now has >2,300 patients enrolled.

Assessment of HCT in Medicare Beneficiaries with MDS

127 centers

1,294 patients ≥65 years old

815 patients 55-64 years old

213 patients <54 years old


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2.2 IMMUNOBIOLOGY RESEARCH PROGRAMThe CIBMTR maintains a Research Repository of paired tissue samples from donors and recipients, both unrelated and related. The Immunobiology Research group manages the Research Repository inventory and immunogenetic testing programs that add critical HLA and killer-cell immunoglobulin-like receptors (KIR) data for use in CIBMTR clinical outcomes studies.

The CIBMTR leverages the NMDP/Be The Match’s investment in the Unrelated Donor Research Repository with the NIH’s investment in the CIBMTR Research Database. Linking outcomes data to immunologic data available in the Research Repository supports studies that include genetic and immunobiologic data and clinical phenotype data.

The Related Donor Research Repository, supported by the Health Resources and Services Administration (HRSA), is a unique opportunity to enhance immunobiologic research. Related donor and recipient samples are better matched than unrelated recipients for HLA, a measure of immunological compatibility, thus reducing the confounding effects of HLA disparity in clinical research.

The combination of the Unrelated Donor and Related Donor Research Repositories facilitates an organized approach to studying transplant biology across the full spectrum of allogeneic HCT.

Program Activities

In 2015, 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks) provided samples to the Research Repository. The Immunobiology Research group enhanced the Research Repository inventory and Immunogenetic Database this year by completing high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient

Research Repository

2,043,345 aliquots

18,901 cell lines

59,375 samples from unrelated donors and 5,128 from related donors

57,018 samples from unrelated recipients and 5,377 from related recipients

10,566 samples from unrelated cord blood units

Samples from complete pairs:

33,919 from complete unrelated adult donor-recipient pairs

4,545 from complete related donor-recipient pairs

3,579 from unrelated cord-recipient pairs

pairs, bringing the total to >17,500 unrelated donor / cord and recipient pairs that have been retrospectively high resolution typed for HLA-A, -B, -C, -DRB1 and -DQB1; >70% include -DPB1, and >10,000 include KIR.

The Immunobiology Research group consolidated all collections (unrelated, related, and clinical trial) under one inventory management system in 2015 and incorporated their information into the Integrated Data Warehouse to facilitate linkage of samples to clinical data. Additionally, the group distributed 8,770 research samples in support of Working Committee studies this year.


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Publications

In 2015, Immunobiology Working Committee study investigators published 6 manuscripts, 4 of which utilized research samples and immunobiology clinical outcomes data. The Immunobiology Research group also supports other investigators’ publications by providing research samples. 9 manuscripts published this year by investigators from other Working Committees and the BMT CTN utilized samples from the Research Repository. A complete list of Working Committee publications is provided in Appendix D1.

Presentations

In 2015, 5 Immunobiology Working Committee study investigators presented at national and international conferences, including 2 oral abstracts at the European Group for Blood and Marrow Transplantation (EBMT) Annual Meeting and 2 posters at the ASH Annual Meeting. A complete list of CIBMTR presentations is provided in Appendix E.

Funding

Support for the Immunobiology Research Program is primarily provided by the Office of Naval Research grant # N00014-14-1-0848, NIH grant # U24CA076518, and HRSA contract # HHSH250201200016C.

The Immunobiology Research Program offers limited research funds supporting immunobiology research studies. The grants are intended to subsidize lab tests, sample collection, or costs associated with the use of research samples. These grants are available to approved CIBMTR studies that support organizational research priorities. For additional information, visit the CIBMTR Grants for Immunobiology Research webpage.

Key Immunobiology Publications this Year

Gadalla SM et al. Association between donor leukocyte telomere length and survival after unrelated allogeneic HCT for severe aplastic anemia. Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. PMC4388056.

Verneris MR et al. HLA mismatch is associated with worse outcomes after unrelated donor reduced-intensity conditioning HCT: an analysis from the CIBMTR. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783-1789. Epub 2015 Jun 6. PMC4568127.

Petersdorf EW et al. High HLA-DP expression and graft-versus-host disease. New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. PMC4560117.

How to Get Involved

All interested parties may attend the annual in-person meeting of the Immunobiology Working Committee at the BMT Tandem Meetings in February. Additionally, the Immunobiology Working Committee encourages highly translational, hypothesis-driven proposals through the Working Committee Study Proposal Review Process (Figure 2.6).

http://www.cibmtr.org/Studies/Immunobiology/Grants/pages/index.aspx

http://www.cibmtr.org/Studies/Immunobiology/Grants/pages/index.aspx


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2.3 CLINICAL TRIALS SUPPORT PROGRAMUsing data from observational studies to support decisions regarding design of prospective clinical trials and / or amendments of such trials, the CIBMTR Research Database provides an important resource to the Clinical Trials Support Program. This program consists of the BMT CTN and RCI BMT, which conduct multicenter clinical trials. Data from the Research Database are used to design, monitor, and analyze these trials.

CIBMTR Coordinating Center Support of Clinical Trials

Trial Planning. Determine patient populations available for trials as well as appropriate inclusion criteria and outcome estimates, and identify transplant centers likely to accrue.

Data Collection. Share data to reduce reporting for transplant centers, and share data collection forms and database structures developed by transplant experts.

Statistical Consultation. Provide expert statistical review of protocols.

Real-Time Accrual Assessment. Review data to evaluate center and patient characteristics of enrolled and non-enrolled patients to address accrual barriers.

Trial Interpretation. Evaluate results of clinical trials, including through the provision of matched controls.

Long-Term Follow-Up Data. Capture follow-up data for long-term or secondary analyses, resulting in considerable cost-savings.

2.3.1 Blood and Marrow Transplant Clinical Trials Network

The BMT CTN, sponsored by NHLBI and NCI, is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation, a contract research organization based in Rockville, MD.

Program Activities

The BMT CTN has launched 37 trials (3 this year), and completed accrual for 28 of these trials (2 this year). The Network has accrued >8,500 patients to its trials from >100 centers, including >1,100 this year. Among trials currently open for enrollment, the BMT CTN achieved an overall accrual rate in 2015 that is approximately 125% of projections. The Network has established a Research Sample Repository that currently includes >350,000 biospecimens. Additionally, the BMT CTN has conducted 48 ancillary and correlative studies.

More detail regarding Network activities and protocols is provided in the annual progress report on the BMT CTN website. A list of Network trials open for enrollment is provided in Appendix G1.

Publications

In 2015, BMT CTN study investigators published 8 manuscripts, 2 of which were primary results papers. These bring the total number of Network publications to 57, including 16 primary results papers. A complete list of 2015 BMT CTN publications is provided in Appendix D2.

https://web.emmes.com/study/bmt2/Progress%20Reports.html



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Key BMT CTN Publications this Year

MacMillan ML et al. A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biology of Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. Epub 2015 Jan 10. PMC4359643.

Khera N et al. Comparison of characteristics and outcomes of trial participants and nonparticipants: example of BMT CTN 0201 trial. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815-1822. Epub 2015 Jun 11. PMC4568172.

Anderlini P et al. Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study. Lancet Haematology. 2015 Sep 1; 2(9):367-375. Epub 2015 Sep 8.

Devine SM et al. Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: results from CALGB 100103 / BMT CTN 0502. Journal of Clinical Oncology. Epub 2015 Nov 2.

Presentations

BMT CTN study investigators presented 11 abstracts (8 oral and 3 poster) at national and international conferences in 2015, two of which were highlighted at the ASH Annual Meeting, one as a Late Breaking Abstract and the other as a 2016 Highlight of ASH. These bring the total number of Network presentations to 59. A complete list of 2015 CIBMTR presentations is provided in Appendix E.

Funding

Support for the BMT CTN Data and Coordinating Center is provided by the NIH grant # U10HL069294 from the NHLBI and NCI.

How to Get Involved

The Network is committed to widespread participation in its trials. If you would like to serve as an Affiliate Center, visit the BMT CTN website for more information. Additionally, you may act as a Center Principal Investigator or champion a trial to increase patient accrual at your Center, serve on a Protocol Team or an Endpoint Review Committee, or act as a Medical Monitor. You may also propose an ancillary study, which uses data, biospecimens, and / or analyses outside the specific objectives of a primary BMT CTN study.

http://bmtctn.net/

http://bmtctn.net/

https://web.emmes.com/study/bmt2/public/Secondaryanalysis.html

https://web.emmes.com/study/bmt2/Researchsample.html


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2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation

The RCI BMT provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting with groups such as the BMT CTN or the national cancer cooperative groups.

Program Activities

The RCI BMT has launched 15 trials, including 3 this year. In 2015, the RCI BMT accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000, of which >21,000 were enrolled in a cohort study examining long-term outcomes of unrelated donors.

The RCI BMT is currently managing 2 Food and Drug Administration (FDA) investigational new drug (IND) protocols for NMDP/Be The Match Operations. Peripheral Blood Stem Cell (PBSC) Procurement accrued >2,100 patients this year, and Cord Blood Access accrued >500. Additionally, the RCI BMT supported 5 studies involving unrelated donor data or sample collection for investigators.

In 2015, the RCI BMT launched two new interconnected software systems to enhance data collection and study management. These systems will increase efficiency, allowing the RCI BMT to effectively support a wide array of clinical trials and research studies.

A complete list of RCI BMT trials is provided in Appendix G2.

Presentations

In 2015, RCI BMT study investigators presented 4 oral abstracts at national and international conferences. A complete list of CIBMTR presentations is provided in Appendix E.

Survey Research Group

The Survey Research Group is a team within the RCI BMT created to assist HCT researchers in developing and conducting research involving questionnaires, direct subject interviews, and patient reported outcomes. The group is responsible for collecting high quality, scientifically valid data from donors, patients, and their families. The Survey Research Group utilizes standardized and semi-structured telephone interviews as well as self-administered questionnaires. In 2015, the Survey Research Group supported 8 active studies and participated in the development of 1 upcoming study.

Funding

Support for the RCI BMT is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID; NMDP/Be The Match; and corporate and private sponsors of specific studies.

The RCI BMT team can work with study investigators to seek funding from a variety of sources, including government agencies, foundations, pharmaceutical companies, and private corporations.

How to Get Involved

Study investigators may solicit clinical trials services from the RCI BMT, including assistance with funding proposals; protocol development and approvals; management of study conduct; data auditing, management, and analysis; and financial administration. Study investigators may also contract for specific services as needed, such as support with surveys, site selection and management, sample management, and more. For additional information, visit the RCI BMT webpage.

http://www.cibmtr.org/Studies/ClinicalTrials/RCI_BMT/pages/index.aspx


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2.4 HEALTH SERVICES RESEARCH PROGRAMHealth services research is the multi-disciplinary field of scientific investigation that studies how social factors, financial systems, organizational structures and processes, technology, and behavior affect treatment outcomes, quality, and cost. The CIBMTR conducts research through the Health Services Research Program in collaboration with NMDP/Be The Match’s Patient and Health Professional Services. Research activities complement those of the CIBMTR’s Health Services and International Studies Working Committee.

Health Services Focus Areas Research

Health Policy

System Capacity Initiative

Program Evaluation

Clinical Trial Support

Training, Education, and Consultation

Program Activities

Research. The Health Services Research Program currently has 9 studies in progress. In 2015, the program completed analysis for 3 research studies.

Health Policy. In collaboration with NMDP/Be The Match’s Payer Policy department, the Health Services Research Program conducted a study, Financial barriers to HCT: the transplant center perspective, to examine the impact of the Patient Protection and Affordable Care Act on access to HCT and treatment decision-making.

Select Health Services Research Studies in Progress

Individualized care plans for HCT survivors

Cost-effectiveness of HCT vs. alternative therapy for acute myeloid leukemia (AML) in patients aged 60-70 years

Analysis of reimbursement for HCT in older patients

HCT multidisciplinary care teams: burnout, moral distress, and career satisfaction

Payer-partnered approach to community-based referral for HCT

Easy-to-read informed consent forms for HCT clinical trials (BMT CTN 1205)

System Capacity Initiative. The goal of the System Capacity Initiative is to determine how the US healthcare system can accommodate the growing number of patients in need of HCT. In 2015, the Health Services Research Program continued to support initiative-related efforts, including the study on HCT workforce burnout.

Program Evaluation. The Health Services Research Program conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs in 2015, including a needs assessment for limited English proficiency patients.


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Clinical Trial Support. A collaboration between the Health Services Research Program and BMT CTN, Easy-to-read informed consent forms for HCT clinical trials evaluates the effectiveness of a novel consent form and describes barriers to implementation. The program also provided technical writing support and developed 3 easy-to-read consent / assent forms and 6 patient information sheets for BMT CTN protocols. Training, Education, and Consultation. In 2015, the Health Services Research Program established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings. More detail regarding program activities is provided in the Health Services Research Annual Report.

Publications

In 2015, Health Services Research study investigators published 4 manuscripts in peer-reviewed journals. A complete list of program publications is provided in Appendix D3.

Presentations

Through the HRSA Office of Patient Advocacy / Single Point of Contact contract, the Health Services Research Program is charged with disseminating findings to all stakeholder groups. In 2015, program investigators presented 3 oral abstracts at the ASH Annual Meeting. A complete list of research-focused CIBMTR presentations is provided in Appendix E.

How to Get Involved

For more information about the Health Services Research Program and / or the HCT Health Economics Interest Group, contact Ellen Denzen, MS, Senior Manager, at [email protected] or 612.884.8562.

Health Services Research Publications this Year

Besse KL et al. Estimating demand and unmet need for allogeneic HCT in the US using geographic information systems. Journal of Oncology Practice. 11(2):e120-e130. Epub 2015 Mar 1. PMC4371120.

Majhail NS et al. National survey of HCT center personnel, infrastructure and models of care delivery. Biology of Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1308-1314. Epub 2015 Mar 31. PMC4466059.

Clauser SB et al. Patient centeredness and engagement in quality-of-care oncology research. Journal of Oncology Practice. 2015 May 1; 11(3):176-179. Epub 2015 Apr 7.

Preussler JM et al. Patient housing barriers to HCT: results from a mixed-methods study of transplant center social workers. Supportive Care in Cancer. Epub 2015 Aug 15.

Funding

Support for the Health Services Research Program is provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID. Individual studies are funded via a variety of mechanisms. Individualized care plans for HCT survivors is supported by the Patient Centered Outcomes Research Institute award # CD-12-12-4062; Easy-to-read informed consent forms for HCT clinical trials is supported by the NHLBI grant # U10HL069294-12S1, and A payer-partnered approach to community-based referral for HCT is supported by the grant # 11762021 from the National Comprehensive Care Network / Pfizer.

http://www.cibmtr.org/Studies/HealthServices/Documents/2014%20HSR%20Annual%20Report%20FINAL%20v1.pdf

http://www.cibmtr.org/Studies/HealthServices/Documents/2014%20HSR%20Annual%20Report%20FINAL%20v1.pdf

mailto:[email protected]


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2.5 BIOINFORMATICS RESEARCH PROGRAMThe Bioinformatics Research Program provides expertise in, and conducts research on, translational and operational bioinformatics.

Program Activities

Current Bioinformatics Research Goals

Develop pipelines to analyze Next Generation Sequencing typing data, including full-gene HLA, KIR, and genome-wide sequencing, to refine our understanding of genetic matching

Investigate the role of genetic ancestry in transplantation, including the best way to match individuals with multiple races in their family tree

Develop data standards and tools for making immunogenetic data portable for research and clinical use

Investigate HLA data from other countries to better understand global frequencies and improve matching

Develop methods for HLA association studies

Publications

In 2015, Bioinformatics study investigators published 9 manuscripts in peer-reviewed journals. A complete list of program publications is provided in Appendix D4.

Presentations

Bioinformatics study investigators presented 11 abstracts (5 oral and 6 poster) at national and international conferences in 2015. A complete list of CIBMTR presentations is provided in Appendix E.

Key Bioinformatics Publications this Year

Slater N et al. Power laws for heavy-tailed distributions: modeling allele and haplotype diversity for the NMDP. PLoS Computational Biology. 11(4):e1004204. Epub 2015 Apr 22. PMC4406525.

Besse K et al. On modeling HLA-identical sibling match probability for alloHCT: estimating the need for an unrelated donor source. Biology of Blood and Marrow Transplantation. Epub 2015 Sep 5.

Buck K et al. 7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry. Biology of Blood and Marrow Transplantation. Epub 2015 Dec 24.

Funding

Support for the Bioinformatics Research Program is primarily provided by the grant # N00014-14-1-0848 from the Office of Naval Research as well as several grants from the NIH.

How to Get Involved

For more information about the Bioinformatics Research Program, visit the NMDP/Be The Match Clinical Bioinformatics webpage or contact Martin Maiers, Director, at [email protected] or 612.627.5892.

https://bioinformatics.bethematchclinical.org/?_ga=1.57959018.1504441593.1429892619

https://bioinformatics.bethematchclinical.org/?_ga=1.57959018.1504441593.1429892619


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2.6 STATISTICAL METHODOLOGY RESEARCH PROGRAMThe CIBMTR has enjoyed a positive, collaborative association with the Division of Biostatistics in the MCW Institute for Health and Society since 1980, an association that is a distinctive asset and crucial to the success of CIBMTR research. This long-standing relationship has many benefits. Biostatisticians ensure the statistical integrity of CIBMTR scientific activities, contribute to results in articles on HCT-related statistical issues for clinical audiences, and support Working Committee study investigators in developing scientific study protocols using CIBMTR data. CIBMTR biostatisticians have pioneered novel methodologic approaches to analyzing HCT data.

Program Activities

HCT is a complex process with multiple competing risks and dramatic changes in the risks of specific events over time. The CIBMTR has developed and evaluated the statistical models used in HCT research and helped guide the research community in appropriate application and interpretation of these sophisticated models.

Statistical Methodology Research Goals

Develop new statistical models

Compare new statistical models with existing solutions using the CIBMTR Research Database

Publications

In 2015, PhD-level biostatisticians in the Statistical Methodology Research Program published 4 peer-reviewed manuscripts and 1 editorial. A complete list of program publications is provided in Appendix D5.

Key Statistical Methodology Publications this Year

Wang T et al. A re-formulation of generalized linear mixed models to fit family data in genetic association studies. Frontiers in Genetics. 6(120):1-10. Epub 2015 Mar 31. PMC4379931.

Eriksson F et al. The proportional odds cumulative incidence model for competing risks. Biometrics. 2015 Sep 1; 71(3)687-695. Epub 2015 May 26. PMC4608382.

Brazauskas R, Logan BR. Observational studies: matching or regression? Biology of Blood and Marrow Transplantation. Epub 2015 Dec 19.

Funding

Support for the Statistical Methodology Research Program is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID and the HRSA contract # HHSH250201200016C.

How to Get Involved

During the BMT Tandem Meetings in February, PhD-level biostatisticians plan and present educational sessions related to statistical design and analysis, and they provide 1:1 statistical consultation to researchers writing proposals or developing protocols for CIBMTR studies. Any interested individual may participate in these sessions. Additionally, the MCW Division of Biostatistics presents a lecture series and a seminar series throughout the year in Milwaukee.

http://www.mcw.edu/Biostatistics.htm

http://www.mcw.edu/Biostatistics/Lecture-Series.htm

http://www.mcw.edu/Biostatistics/Events/Seminar-Series.htm


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2.7 STEM CELL THERAPEUTIC OUTCOMES DATABASE (SCTOD)The CIBMTR administers the SCTOD for the HRSA-sponsored C.W. Bill Young Cell Transplantation Program, established by the Stem Cell Therapeutic and Research Act of 2005. Continued support for the SCTOD is provided through the Stem Cell Therapeutic and Research Reauthorization Act of 2010.

C.W. Bill Young Cell Transplantation Program Goals Fulfilled by the SCTOD

Collect, analyze, and report outcomes data for all allogeneic transplants and other therapeutic uses of blood stem cells

Publicize information about HCT to patients, families, health care professionals, and the public

Define better processes for identifying unrelated matched marrow donors, PBSC donors, and cord blood units through one electronic system

Increase availability of unrelated adult volunteer donors and cord blood units

Expand research to improve patient outcomes

Program Activities

Annually, the CIBMTR publishes HCT volumes and performance data by transplant center and provides public access to this information via the HRSA Blood Cell Transplant website.

Center-Specific Volumes and Survival Analysis. As part of the contract to operate the SCTOD, the CIBMTR provides the annual volume of transplants performed at each center and performs a center-specific survival analysis comparing the one-year survival rates among US centers. The report assesses transplants from both related and unrelated

SCTOD Contract Requirements

Collect HCT outcomes data for:

• All allogeneic HCTs performed in the US using related or unrelated donors

• All allogeneic HCTs worldwide that use grafts procured through the C.W. Bill Young Cell Transplantation Program

• Clinical applications of hematopoietic stem cells other than hematopoietic cell recovery

Use the data collected for the SCTOD to evaluate the performance of transplant centers

Provide specific SCTOD data to the public

Collect a basic set of data for analyses of program use, center-specific outcomes, donor registry, cord blood inventory size, and patient access to HCT

Establish a Related Donor-Recipient Research Sample Repository (Section 2.2)

donors. The most recent report was finalized in September 2015 and contains information on all first allogeneic transplants performed in US centers from January 1, 2011, through December 31, 2013.

Center Outcomes Forums. The CIBMTR has conducted 4 Center Outcomes Forums to engage relevant stakeholders in the center-specific outcomes reporting process. The most recent meeting was held in June 2014 and generated recommendations to improve risk adjustment, enhance future collection of relevant data elements, and develop tools to enhance centers’ quality improvement efforts.

http://bloodcell.transplant.hrsa.gov/

http://www.cibmtr.org/Meetings/Materials/CSOAForum/pages/index.aspx


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Quality of Life Assessments. In 2011, the CIBMTR launched a pilot program at 5 adult and 3 pediatric transplant centers. The principal goal was to test center-based recruitment of patients to a quality-of-life data collection program, followed by communication between the CIBMTR and the patient to collect longitudinal quality-of-life information. This pilot program is unique in that the CIBMTR collects data directly from patients after HCT. Accrual closed in 2013 with 390 patients enrolled. Data analysis is in progress.

Cellular Therapies for Regenerative Medicine (CTRM). The CTRM data repository tracks novel uses of blood stem cells. The SCTOD contract mandates data collection on uses of cells found in bone marrow, peripheral blood, and umbilical cord blood for alternative therapeutic applications, including regenerative medicine.

The CTRM data repository captures uses of cells for the treatment of diseases without the intention of replacing the recipient’s hematopoietic function. These therapies include, but are not limited to, treatment of malignancies as well as infectious, cardiovascular, rheumatologic, neurologic, musculoskeletal, and endocrinologic diseases with the intent to improve organ function. As of November 30, the CTRM data repository includes data for 983 patients.

The CIBMTR anticipates the expansion of the cellular therapy field with the use of not only hematopoietic derived cells but also cells from other tissues; therefore, it is expanding the capability and flexibility of data collection in this rapidly changing area. In October, the CIBMTR held a Cellular Therapy Forum (Section 2.1.2) to discuss how to further develop its cellular therapy registry.

Study Summaries for Patients. In conjunction with Be the Match’s Patient and Health Professional Services and the Consumer Advocacy Committee, the CIBMTR publishes lay summaries of CIBMTR publications for patients and their loved ones. In 2015, the CIBMTR published 12 patient-friendly research summaries.

Funding

Support for the SCTOD is provided by the HRSA contract # HHSH250201200016C.

How to Get Involved

All US centers performing allogeneic HCTs provide data to the CIBMTR for the SCTOD. These data are used to generate reports, which are distributed to transplant center medical directors and posted on the HRSA Blood Cell Transplant website.

Publicly Available Reports developed by the CIBMTR for the SCTOD

Transplant Outcomes

• US Patient Survival Report

• US Transplant Data by Center Report

• US Transplant Data by Disease Report

Transplant Activity Report

These reports are available on the HRSA Blood Cell Transplant website



http://bloodcell.transplant.hrsa.gov/research/transplant_data/us_tx_data/index.html

http://bloodcell.transplant.hrsa.gov/research/transplant_data/us_tx_data/survival_data/survival.aspx

http://bloodcell.transplant.hrsa.gov/research/transplant_data/us_tx_data/data_by_center/center.aspx

http://bloodcell.transplant.hrsa.gov/research/transplant_data/us_tx_data/data_by_disease/national.aspx

http://bloodcell.transplant.hrsa.gov/research/transplant_data/transplant_activity_report/index.html

http://bloodcell.transplant.hrsa.gov/research/transplant_data/index.html



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2.8 CORPORATE PROGRAMThe CIBMTR Corporate Program provides opportunities for industry collaborators to access CIBMTR data and statistical support to address questions specific to their business needs through Corporate Membership as well as Corporate Studies and Projects.

Corporate Membership. The CIBMTR Corporate Membership program provides a variety of resource materials to corporations needing access to the most current and comprehensive data on HCT. These materials are useful for Marketing Managers, Medical Directors, Research Directors, Product Managers, Case Managers, and Transplant Coordinators. There are four Corporate Membership levels available, each described on the CIBMTR Corporate Membership Program webpage.

Corporate Membership Benefits

CIBMTR Report on Survival Statistics for BMT

Center Volumes Dataset

Worldwide CIBMTR Directory of BMT Physicians

Reduced registration rates at CIBMTR meetings and educational forums, including the BMT Tandem Meetings

Access to CIBMTR data and resources

Corporate Studies and Projects. Corporate partners may contract with the CIBMTR to conduct a study, support a project involving more complex analyses, or license a specified data set. Organizations interested in funding a study, such as one comparing HCT with one or more non-HCT therapies, or using historical controls, can negotiate with the CIBMTR for data and / or expert statistical analyses.

Program Activities

The CIBMTR engaged in 8 studies with corporate partners in 2015. Currently 14 organizations participate in the CIBMTR Corporate Membership program, including 9 that joined this year.

How to Get Involved

If you would like to learn more about the CIBMTR Corporate Program, visit the CIBMTR Corporate Membership webpage or contact Sherry Fisher, Director of Advancement, at [email protected] or 414.805.0687. If you are a Corporate Member requesting analyses, please complete the Corporate Member Information Request Form.

http://www.cibmtr.org/support/corporate/corporatemembership/Pages/index.aspx





http://www.cibmtr.org/Data/Request/Pages/Corporate-Member.aspx

http://www.cibmtr.org/Data/Request/Pages/Corporate-Member.aspx

CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE

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3.0 HOW WE SHARE KNOWLEDGE

The CIBMTR is committed to sharing the data we collect as well as the information and knowledge produced from our data and our extensive collaborations with investigators in the HCT field.

The CIBMTR shares its knowledge in different ways. To determine the best way to access specific types of CIBMTR knowledge, review Figure 3.1 and Tables 3.2-3.5.

Figure 3.1. How to Access CIBMTR Knowledge

Details provided in Tables 3.2-3.5.


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Table 3.2. How to Access CIBMTR Information*

Information

GENERAL In addition to reviewing this report, access the Summary of Accomplishments on the Administrative and Progress Reports webpage. Read editions of the quarterly newsletter on the Newsletters webpage, and email cibmtr-news@mcw. edu to be added to the electronic distribution list.

ACTIVITIES Review Section 2 or visit the What We Do webpage. Visit the Studies webpage, SCTOD webpage, or Corporate Support webpage to learn more about CIBMTR research programs, the SCTOD, or Corporate Program, respectively.

PUBLICATIONS Review the CIBMTR’s >1,000 publications on the Publication List webpage. For lay summaries of selected CIBMTR publications, written specifically for patients and the lay public, visit the Study Summaries for Patients webpage.

MEETINGS Visit the BMT Tandem Meetings webpage to view agendas, register, reserve housing, and submit abstracts.

TRAINING Review Section 3.4 or visit the Training and Reference webpage to access the Center Reference Guide, Forms Instruction Manual, FormsNet and AGNIS trainings, and online courses.

OTHER Email [email protected].

Table 3.3 How to Access CIBMTR Data*

Data

TYPES Review the baseline and follow-up data available for recipients and donors on the Types of Data Available for Research or Request webpage.

STANDARD REPORTS Access the Summary Slides, BMT Survival Statistics Report, Center Transplant Activity Report, Patient Transplant Outcomes Reports, and Center-Specific Survival Reports via Table 3.6 or on the Slides and Reports webpage.

ELECTRONIC RETURN OF CENTER Utilize the Data Back to Centers application on the Portal site to download TED-level variables that have been validated and processed in the CIBMTR Research Database.

RESEARCH STUDY Propose a study as explained on the How to Propose a Study webpage, or participate in one of the existing studies listed on the Working Committee Study Lists webpage.

CORPORATE ANALYSIS Complete the Corporate Member Information Request Form.

CUSTOM ANALYSIS Complete the Custom Information Request Form.


*If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).

http://www.cibmtr.org/About/ProceduresProgress/Pages/index.aspx


http://www.cibmtr.org/ReferenceCenter/Newsletters/Pages/index.aspx


mailto:ailto:cibmtr-news@mcw?subject=I%20want%20to%20be%20added%20to%20the%20newsletter%20distribution%20list

mailto:ailto:cibmtr-news@mcw?subject=I%20want%20to%20be%20added%20to%20the%20newsletter%20distribution%20list

http://www.cibmtr.org/About/WhatWeDo/Pages/index.aspx

http://www.cibmtr.org/About/WhatWeDo/Pages/index.aspx

http://www.cibmtr.org/Studies/Pages/index.aspx

http://www.cibmtr.org/About/WhatWeDo/SCTOD/Pages/index.aspx

http://www.cibmtr.org/Support/Corporate/Pages/index.aspx

http://www.cibmtr.org/Support/Corporate/Pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/PubList/Pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/Patient/PatientSummaries/pages/index.aspx

http://www.cibmtr.org/Meetings/Tandem/Pages/index.aspx

http://www.cibmtr.org/Meetings/Tandem/Pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/Pages/index.aspx


http://www.cibmtr.org/DataManagement/TrainingReference/Documents/CtrRefGuide.pdf

http://www.cibmtr.org/manuals/fim


http://www.cibmtr.org/DataManagement/TrainingReference/FormsNet/pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/AGNIS/pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/eLearning/Pages/index.aspx


http://www.cibmtr.org/Data/Available/pages/index.aspx

http://www.cibmtr.org/Data/Available/pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/SlidesReports/SummarySlides/pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/SlidesReports/StatReport/Pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/SlidesReports/StatReport/Pages/index.aspx





https://bethematch.org/For-Patients-and-Families/Getting-a-transplant/Choosing-a-transplant-center/U-S--transplant-centers/


http://www.cibmtr.org/ReferenceCenter/SlidesReports/Pages/index.aspx

https://portal.cibmtr.org/



http://www.cibmtr.org/Studies/Observational/StudyLists/Pages/index.aspx

http://www.cibmtr.org/Studies/Observational/StudyLists/Pages/index.aspx

http://www.cibmtr.org/Data/Request/Documents/Corporate_Member_Information_Request_Form.pdf

http://www.cibmtr.org/Data/Request/Documents/Corporate_Member_Information_Request_Form.pdf

http://www.cibmtr.org/Data/Request/Documents/Custom_Information_Request_Form.pdf



https://www.cibmtr.org/pages/index.aspx


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Table 3.4 How to Access CIBMTR Tools*

Tools

DISEASE RISK INDEX ASSIGNMENT TOOL

Access this tool on the DRI Assignment Tool webpage to categorize patients undergoing allogeneic HCT for hematologic malignancy by disease risk.

PATIENT ONE-YEAR SURVIVAL CALCULATOR FOR ALLOGENEIC TRANSPLANTS

Transplant Center Medical Directors may access this tool on the Portal site to predict one year survival for individual allogeneic HCT recipients.

Table 3.5 How to Access CIBMTR Biospecimens*

Biospecimens

SAMPLES TYPES AND INVENTORY Determine the >2 million samples available in the Research Repository via the Sample Types and Inventory Summary webpage.

REQUESTING SAMPLES For studies that include recipient clinical outcome data, propose a study as explained on the How to Propose a Study webpage. For studies that do not include clinical outcome data, review the How to Request Samples from the Research Sample Repository webpage.



http://www.cibmtr.org/referencecenter/statistical/tools/pages/dri.aspx

http://www.cibmtr.org/referencecenter/statistical/tools/pages/dri.aspx


http://www.cibmtr.org/Samples/Inventory/Pages/index.aspx

http://www.cibmtr.org/Samples/Inventory/Pages/index.aspx



http://www.cibmtr.org/Samples/Requests/Pages/index.aspx






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Table 3.6. Standard Reports Published by the CIBMTR

Report Title Description Month(s) Released Format Accessibility*

CIBMTR Summary Slides

Charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT; developed in conjunction with the BMT Tandem Meetings

February PPT Published on the CIBMTR Summary Slides webpage

CIBMTR Progress Report

Information on the CIBMTR's goals and achievements as well as operational details on how the CIBMTR is funded, supported, promoted, and maintained

February PDF Published on the CIBMTR Administrative and Progress Reports webpage

CIBMTR Report of Survival Statistics for BMT

Highly detailed report on survival statistics that describes use and outcome of autologous and allogeneic HCT in the >500 centers that have participated in the CIBMTR

September Word Via Corporate Membership Program (Section 2.8) or by request from physicians for making treatment decisions or clinical investigators planning clinical studies to [email protected]

CIBMTR Summary of Accomplishments

High level summary of CIBMTR fiscal year accomplishments and high impact publications

September PDF Published on the CIBMTR Administrative and Progress Reports webpage

US Centers Annual Transplant Activity Report

Dataset containing center-specific pre-transplant patient-, disease-, and transplant-related characteristics data for nearly all allogeneic and a majority of autologous HCTs performed in the US annually since 2008

September PDF Published on the HRSA Blood Cell Transplant website

US Patient Center-Specific Survival Report

Comparison of observed to expected one-year survival rates among centers in the C.W. Bill Young Cell Transplantation Program network; evaluates outcomes for transplants using both related and unrelated donors

December Web Published on the Be The Match Transplant Center Directory webpage; available as a Word document upon request to [email protected]














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Report Title Description Month(s) Released Format Accessibility*

US Patient Transplant Outcomes

Disseminated in 3 different reports: • US Patient Survival Report: 100-day, 1-

year, and 3-year survival rate estimates for US HCT recipients by disease and donor type

• US Transplant Data by Center Report: Number of bone marrow and cord blood transplants performed at a specific transplant center

• US Transplant Data by Disease Report: Number of bone marrow and cord blood transplants for a specific disease

December Web Published on the HRSA Blood Cell Transplant website

US Allogeneic Transplant Activity Report

Report containing patient, disease, donor HLA match, donor age, and gender match information for allogeneic transplant activity in the US since 2010

January April July

October

PDF Via Corporate Membership Program (Section 2.8)

CIBMTR Newsletter Articles regarding Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community

February May

August November

Web Published on the CIBMTR Newsletters webpage and distributed via email; contact [email protected] to be added to the distribution list

Study Summaries for Patients

Summaries of CIBMTR research publications written for patients and others in the lay public

Ongoing PDF Published on the CIBMTR Study Summaries webpage











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3.1 INFORMATION REQUEST SERVICEThe CIBMTR Information Request Service provides timely access to data on transplantation to patients, physicians, hospitals, pharmaceutical companies, insurance companies, and others involved in healthcare. Requests range from simple queries of patient, disease, and transplant frequencies to those with greater complexity involving specific data combinations and / or statistical analysis of outcomes.

Potential Reasons for Information Requests

Self-education and decision making

Patient counseling or clinical decision making

Presentation support

Transplant center assessments

Clinical trial planning

Market assessments

Coordinating Center staff members fulfill requests related to clinical decision making within three days and most other requests within three weeks. If a request will take more than an estimated four weeks to fulfill, a Coordinating Center staff member will contact the requestor to discuss an appropriate timeline.

Accomplishments

In 2015, the CIBMTR fulfilled 469 requests for information and data (Table 3.7).

Table 3.7. Data Requests Addressed by the CIBMTR in 2015

Requestor Number of Requests

Physician / Researcher 352

Patient or Relative 41

Pharmaceutical / Biotech Company 38

Market Research Firm 22

Student 6

Patient Advocacy Group 4

News Media 4

Federal Government Agency 1

Cord Blood Bank 1

TOTAL 469

How to Access

For more information about requesting data from the Research Database, visit the CIBMTR How to Request Data webpage. If you would like a one-time, custom analysis, complete the Custom Information Request Form. If you have questions about requesting CIBMTR data, please contact [email protected].

http://www.cibmtr.org/Data/Request/Pages/index.aspx

http://www.cibmtr.org/Data/Request/Pages/index.aspx




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3.2 INTERNET PRESENCEThe CIBMTR Internet presence provides the scientific community and the public with access to HCT information. Current websites include general information about HCT and CIBMTR activities; training and support; a shared communications and collaborative environment for member centers; and secure web access to CIBMTR data for Working Committees, transplant centers, scientific investigators, and CIBMTR staff.

3.2.1 CIBMTR Public Website

The CIBMTR public website (cibmtr.org) is unrestricted and provides information about the CIBMTR and its research. It supports the Working Committees and BMT CTN with information regarding proposal submission, access to a listing and summaries of all studies in process, and access to a summary of all CIBMTR publications. The website facilitates data and information requests, and it provides access to all current and past data collections forms, training manuals, and videos as well as other materials for both investigators and data professionals. The website information is, in part, supported by DISCO (Data and Information for Statistical Center Operations), an application which maintains data on >750 studies, >1,000 publications, and >1,800 authors and their institutions at time of publication. In 2015, the CIBMTR public website had approximately 418,000 unique page views.

About CIBMTR

Administrative and Progress Reports (663 unique page views in 2015) Provides access to the CIBMTR’s annual Progress Report, annual Summary of Accomplishments, and Manual of Operations.

Studies

Working Committee Studies Lists (4,835 unique page views in 2015) A summary of the planned, in-progress, and recently published clinical outcomes studies for each Working Committee.

Meetings

Annual BMT Tandem Meetings Materials (34,073 unique page views in 2015) Provides access to agendas, handouts, and educational materials from specific meetings at the BMT Tandem Meetings: Working Committee Meetings and Clinical Research Professionals / Data Management Conferences.

Reference Center

Summary Slides (10,654 unique page views in 2015) Includes charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT.

Web-based US Transplant Reports (31,964 unique page views in 2015) Directs users to the Be The Match US Center Listing Report and customizable reports of patient survival and transplant available through the HRSA Blood Cell Transplant website.

Publication List (5,836 unique page views in 2015) Searchable descriptive list of >1,000 publications resulting from the use of CIBMTR data and statistical resources.

http://www.cibmtr.org/


http://www.cibmtr.org/Studies/Observational/StudyLists/pages/index.aspx

http://www.cibmtr.org/Meetings/Materials/Pages/index.aspx


http://www.cibmtr.org/ReferenceCenter/SlidesReports/USStats/pages/index.aspx





http://www.cibmtr.org/ReferenceCenter/PubList/Pages/index.aspx


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Newsletter (3,199 unique page views in 2015) Published 3-4 times per year. Articles feature updates on Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community.

Patient Resources (4,590 unique page views in 2015) Includes lay summaries of CIBMTR research articles as well as post-transplant care recommendations for adult and pediatric autologous and allogeneic HCT recipients to help patients and clinicians understand and plan for the specialized care of transplant recipients. 12 lay summaries were published in 2015.

Statistical Resources (4,183 unique page views in 2015) Provides access to resources offered through the unique partnership between the CIBMTR and MCW Division of Biostatistics, including biostatistical publications, a series of statistical lectures targeted at basic and clinical investigators, and research tools, such as the Disease Risk Index Assignment Tool.

Data Management

Data Management Manual (38,071 unique page views in 2015) A comprehensive reference document for completing CIBMTR data collection forms. The manual also details reporting requirements, describes protocols and the consent process, and includes downloadable versions of report forms.

Data Back to Centers Application (628 unique page views) Links to the Data Back to Centers (DBtC) application that provides CIBMTR member centers the ability to retrieve all the TED-level data their center has reported to the CIBMTR through FormsNet or AGNIS.

Data Collection Forms (47,303 unique page views in 2015) Provides access to current and retired versions of the forms used by the CIBMTR to collect standard data elements for all transplant recipients.

Training and Reference (68,004 unique page views in 2015) Provides access to a wide variety of CIBMTR data management training and reference materials.

3.2.2 CIBMTR Collaborative Site

The CIBMTR Collaborative site (collaborate.cibmtr.org) uses the SharePoint Enterprise Collaboration platform to promote cooperative work among CIBMTR staff members and provides a communication platform for specific studies and initiatives. This site is secured by username and password, and user-specific security credentials are assigned centrally.

The CIBMTR uses the site for storing and sharing protocol and consent documents, donor / recipient tracking tools, confidential committee information, data, manuscript drafts, and other relevant information. While only two Working Committees currently use the Collaborate site on a regular basis, it is available to all Working Committees for sharing information.


http://www.cibmtr.org/ReferenceCenter/Patient/Pages/index.aspx



http://www.cibmtr.org/ReferenceCenter/Patient/Guidelines/pages/index.aspx



http://www.cibmtr.org/ReferenceCenter/Statistical/Pages/index.aspx

http://www.cibmtr.org/ReferenceCenter/Statistical/Tools/Pages/DRI.aspx


http://www.cibmtr.org/DataManagement/AdminResources/DBtC/pages/index.aspx

http://www.cibmtr.org/DataManagement/DataCollectionForms/Pages/index.aspx


https://collaborate.cibmtr.org/


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3.2.3 CIBMTR Portal Site

The CIBMTR Portal site (portal.cibmtr.org) delivers applications and data to CIBMTR transplant centers and other partners. In 2015, unique, external visitors viewed 2,675 portal pages. Four applications are currently hosted on this site:

Data Back to Centers

The current DBtC application provides authorized users the ability to download CIBMTR TED-level data variables for their centers. The data have been validated and processed in the CIBMTR Research Database and are reviewed and refreshed quarterly. Legacy International Bone Marrow Transplant Registry (IBMTR) data from as far back as 1964 and some legacy NMDP/Be The Match data from as far back as 1987 are available. In 2015, 628 unique, non-CIBMTR visitors viewed 914 DBtC pages and downloaded data 418 times. In Spring 2016, the CIBMTR plans to release an enhanced DBtC (eDBtC) application that will give centers access to both TED- and CRF-level source data as well as a variety of analytic tools via a user-friendly application called Qlikview.

Center Volumes Portal

The Center Volumes Portal allows centers to preview; correct, if necessary; and approve center volume data published annually to the HRSA Blood Cell Transplant website. The CIBMTR gives centers access to display and download the previous six years (2009-2014) of volume data as well as the current year under review (2014). In 2015, 188 unique, external visitors viewed 706 Center Volumes Portal pages.

Patient One-Year Survival Calculator – Allogeneic Transplants

Accessible by medical directors, the Patient One-Year Survival Calculator for Allogeneic Transplants provides centers with a tool to predict one year survival for individual allogeneic HCT recipients. The calculator data are updated annually to reflect new information contained in the center outcomes analysis. In 2015, 448 unique, non-CIBMTR visitors viewed 1,357 survival calculator pages.

Qlikview

In 2015, the CIBMTR selected Qlikview, a third party business intelligence tool, to provide transplant centers with self-service access to their data and information, including descriptive statistics, outcomes, and trends. Using Qlikview, centers will be able to more readily visualize their data in predefined dashboards, interact with their data through ad hoc analytics, and export their data to files that will support further analysis. The CIBMTR will introduce two Qlikview applications at the 2016 BMT Tandem Meetings: eDBtC, which will provide transplant centers with access to their TED- and CRF-level data, and Center-Specific Analysis Tool, which will provide transplant centers with access to their SCTOD database data.




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3.2.4 Be The Match Public Website

The Be The Match Public website (bethematch.org) is designed for patients and families, donors, and supporters. It incorporates detailed information about transplantation and donation written for the public. The website provides scientific information in lay terms for donors related to the donation process and for patients related to specific diseases, various treatment options, the process of transplantation, and life after transplant. It also addresses concerns related to specific populations, including children and caregivers. The CIBMTR collaborates with NMDP/Be The Match to provide content for several areas of this website, including data for the US Center Listing Report.

US Center Listing Report

Transplant Center Directory Provides transplant center specific information about facilities, personnel, diseases treated, cost, and transplant experience, including the number of transplants performed and survival rates by age, disease type, and disease stage.

3.2.5 Be The Match Clinical Website

The Be The Match Clinical website (bethematchclinical.org) is designed for clinicians, network participants, payors, and bioinformatics professionals. For clinicians, the website provides access to evidence-based tools, clinical guidelines, outcomes data, and education courses on HCT. The website also provides information specific to types of network participants: transplant centers, donor centers, apheresis and collection centers, and cord blood banks. For payors, the website offers information to help individuals understand BMT, determine coverage, and answer employer and patient questions. Related to bioinformatics, the website provides resources for immunogenetic-focused research and operational bioinformatics as well as frequently used HLA tools.

3.2.6 HRSA Blood Cell Transplant Website

The HRSA Blood Cell Transplant website (bloodcell.transplant.hrsa.gov) provides information for the public, physicians, and other constituents. It incorporates transplant resources, donor information, and cord blood information as well as research, data, and outcomes. CIBMTR data and research findings are incorporated in numerous ways, including through CIBMTR-created reports:

Transplant Outcomes and Data

US Patient Survival Report Provides disease-specific post-HCT survival estimates by the length of time after transplant: 100 days, 1 year, and 3 years. Survival estimates are also available by patient age, patient gender, patient race, or cell source.

Transplant Data by US Center Report Displays the number of adult donor and cord blood transplants performed at a specific transplant center.

Transplant Data by Disease Report Displays the number of adult donor and cord blood transplants reported for a specific disease. Totals are also available by patient age, patient gender, patient race, cell source, and the year the transplant was performed.

Transplant Activity Report Displays the number of transplants performed at US transplant centers, including autologous as well as related and unrelated allogeneic. Numbers are also available by patient age, patient gender, patient race, cell source, disease, transplant center location by state, and year in which the transplant was performed.

http://www.bethematch.org/


http://www.bethematchclinical.org/


http://bloodcell.transplant.hrsa.gov/RESEARCH/Transplant_Data/US_Tx_Data/Survival_Data/survival.aspx

http://bloodcell.transplant.hrsa.gov/RESEARCH/Transplant_Data/US_Tx_Data/Data_by_Center/center.aspx

http://bloodcell.transplant.hrsa.gov/RESEARCH/Transplant_Data/US_Tx_Data/Data_by_Disease/national.aspx



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3.2.7 Other Applications and Data Exchange Standards

The CIBMTR has 7 methods for sharing data. The 4 hosted on the CIBMTR Portal site (DBtC, Center Volumes Portal, Patient One-Year Survival Calculator, and Qlikview) were described in Section 3.2.3. The other 3 methods for sharing data are:

AGNIS

AGNIS (A Growable Network Information System) allows participating centers to electronically collect and share data with the CIBMTR as well as others who link to AGNIS. Data are entered once and then distributed and synchronized among databases. In 2015, a total of 12,873 forms for 1,840 patients were submitted through AGNIS by 20 US transplant centers and by EBMT for 47 of their affiliated centers.

BRIDG

The BRIDG (Biomedical Research Integrated Domain Group) Model is an information model, representing a shared view of the concepts of basic, pre-clinical, clinical, and translational research. Common data elements for certain standard CIBMTR forms have been extracted and associated in the BRIDG model to one of three contexts: recipient, donor, or stem cell product. Future expansion of the BRIDG model will add HCT content as the basis for a physical database model, which will help remove barriers that transplant centers experience in electronic transfer of HCT data to the SCTOD. It will provide a foundation upon which centers and vendors can develop their own in-house data systems and, eventually, develop electronic medical record integration engines to submit data to the CIBMTR Research Database.

Disease Risk Index Assignment Tool

In March 2015, the CIBMTR launched a Disease Risk Index Assignment Tool developed by investigators at the Dana Farber Cancer Institute and validated in a large CIBMTR study. It is intended for use by clinical researchers. The tool was developed for the primary outcome of overall survival after HCT and, at present, only applies to adult patients with hematologic malignancies. It is NOT intended to give an accurate prognosis for individual patients. In 2015, 1,370 unique, non-CIBMTR visitors viewed the Disease Risk Index Assignment Tool 3,764 times.



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3.3 BMT TANDEM MEETINGSThe BMT Tandem Meetings are co-sponsored with the ASBMT and are held annually in February. They include 5 days of plenary sessions, concurrent scientific sessions, and other meetings. Reports on recent progress and updates in basic science, translational research, and clinical studies are targeted to worldwide physicians, scientists, and other health professionals with an interest in HCT.

2015 BMT Tandem Meetings

With 3,132 attendees from 47 countries, the 2015 BMT Tandem Meetings included 5 plenary sessions, 11 concurrent sessions, 108 oral abstracts, 2 poster sessions, 6 corporate-supported symposia, and 3 product theaters. Continuing Medical Education (CME) and Continuing Education credits were issued through MCW to physicians and allied health professionals. In addition to the extensive scientific agenda, many educational opportunities focused on young investigators and other allied health professionals.

Clinical Research Professionals / Data Management Conference With almost 200 attendees, this conference provided forms training, which increases the accuracy with which CIBMTR forms are completed.

BMT CTN Coordinators and Investigators Meetings With approximately 100 and 300 attendees, respectively, these meetings focused on treatment options; study management, such as promoting studies and reporting adverse events; processes, such as endpoint review; and specific clinical trials.

IT Forum With almost 100 attendees, this forum discussed the Research Data Life Cycle and various ways to share data, including from electronic medical records via BRIDG.

BMT Center Administrators Conference With approximately 165 attendees, this conference focused on many topics related to quality and outcomes, including benchmarking and key metrics, such as readmission rates, as well as Medicare coverage and workforce capacity.

BMT Pharmacists Conference With almost 200 attendees, this conference presented the latest research and best practices with a focus on specific diseases and preparative regimens.

Transplant Nursing Conference With almost 500 attendees, this conference presented the latest research as well as disease-specific information and communication techniques.

BMT Clinical Education Conference This conference is designed for Nurse Practitioners, Physician Assistants, Fellows, and Junior Faculty. With almost 200 attendees, the conference focused not only on the latest clinical research but also informed consent and mental health.

2016 BMT Tandem Meetings

The 2016 BMT Tandem Meetings will include 5 plenary sessions, 9 concurrent sessions, 133 oral abstracts, 2 poster sessions, 5 corporate-supported symposia, and 5 product theaters. They are expected to attract approximately 3,200 attendees.


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3.4 DATA MANAGEMENT TRAININGThe CIBMTR has developed comprehensive, secure and efficient applications to allow you to electronically submit data to the CIBMTR. Visit the CIBMTR Data Management Training and Reference webpage to access resources.

Center Reference Guide Learn about participation in CIBMTR research, center membership, access to FormsNet, data manager education, mentor program, forms submission process and many useful tips and links.

Manuals Find the answers to your data submission questions by accessing the Forms Instructions Manual, which includes general instructions and instructions for each form type.

FormsNet

Learn how to submit data to the CIBMTR via the FormsNet application, a secure clinical research management system, which is in compliance with SCTOD requirements.

Conference Materials Access meeting materials as well as audio and visual presentations on the form submission process presented at Clinical Research Professionals / Data Management Conferences.

AGNIS Learn how to retrieve and transmit form data, extracted directly from your own institution’s database, directly to the FormsNet application using AGNIS, a secure, standards-based system.

Legacy Data Review retired data manuals, forms, and other archived documents for reference purposes and to assist in making changes to legacy data.

Adverse Events Learn how to report adverse events and product issues through FormsNet.

Newsletters and eBlasts Read archived issues of the Data Matters Training Newsletter and eBlasts.

Tip Sheets Access sheets providing tips and instructions for various CIBMTR forms.

Online Training Review educational modules developed for new and seasoned data managers through the NMDP/Be The Match Learning Center.

Learning Center Courses

Data Back to Centers Overview

HLA SERIES

Introduction to HLA

HLA Reporting (Form 2005)

Basic Biology of HLA

Advanced Biology of HLA

ESSENTIAL FORM SERIES

Baseline Form 2000

CRID 2804

Form 2400 Pre-TED

Indication for CRID Assignment (Form 2814)

Infusion Data (Form 2006) Reporting Overview

Reporting Preparative Regimen on Pre-TED (Form 2400) and Baseline (Form 2000)

DISEASE SPECIFIC SERIES

Form 2016 / 2116 Plasma Cell Disorders

Form 2018 / 2118 Lymphoma

http://www.cibmtr.org/DataManagement/TrainingReference/pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/Documents/CtrRefGuide.pdf

http://www.cibmtr.org/DataManagement/TrainingReference/Manuals/pages/index.aspx



http://www.cibmtr.org/DataManagement/TrainingReference/FormsNet/pages/index.aspx

http://www.cibmtr.org/Meetings/Materials/CRPDMC/Pages/2015Index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/AGNIS/pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/LegacyData/pages/index.aspx

http://www.shorewest.com/homes/1http:/www.cibmtr.org/DataManagement/TrainingReference/Pages/AdverseEvents.aspx

http://www.shorewest.com/homeshttp:/www.cibmtr.org/DataManagement/TrainingReference/DataMattersTrainingNewsletters/Pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/FAQs/Pages/index.aspx

http://www.cibmtr.org/DataManagement/TrainingReference/eLearning/Pages/index.aspx

https://network.bethematchchclinical.org/learningcenter

CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA

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4.0 HOW WE COLLECT AND MANAGE DATA

4.1 RESEARCH DATA LIFE CYCLEThe Research Data Life Cycle (Figure 4.1) describes the path of data from the point of capture to its ultimate use in analysis, reporting, and publication.

The process begins with data collection. Most centers enter data in FormsNet, a web application now in its third generation. Centers that have implemented local or third party systems can also capture and submit data electronically using AGNIS. An overriding goal of these applications is to ease the data capture burden on the centers.

Following collection, data undergo quality assessment and validation and are extracted 4 times per month and loaded into the CIBMTR Research Database.

Data Sharing completes the cycle, providing data for analysis that have been collected and curated to ensure research value. These data are extracted from the Research Database in monthly and quarterly retrievals to serve a range of research and stakeholder needs. The data retrievals provide the basis for research study data files, reports, and externally-requested datasets. TED-level data are also directly available to centers through use of the DBtC application (Section 3.2.3).

Figure 4.1. Research Data Life Cycle

Abbreviations: AGNIS = A Growable Network Information System, CRID = CIBMTR Recipient Identification Number, CRF = Comprehensive Report Form, FN = FormsNet, IDW = Integrated Data Warehouse, RDB = Research Database


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4.2 COLLECTING AND STORING DATA

4.2.1 FormsNet

More than 95% of data collected by the CIBMTR is submitted electronically via FormsNet, a comprehensive electronic data submission system containing >240 forms related to capturing HCT outcomes for donors and recipients. The application was updated in 2015 to provide key enhancements supporting operational efficiencies and enhanced data collection. For instance, a more flexible CIBMTR Recipient ID (CRID) Assignment Form establishes a patient ID independent of treatment, allowing the CIBMTR to expand data collection to patients who receive cellular therapy as well as non-HCT treatments. In addition, the CIBMTR released several forms to support data collection needs of the Radiation Injury Treatment Network (RITN), which will collect incident, medical history, and follow-up data on casualties exposed to or contaminated by radioactive material in the event of a national radiation event.

4.2.2 Research Database

The CIBMTR Research Database now contains information on >425,000 patients. Submission of outcomes data is mandatory for allogeneic HCTs in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous HCTs are reported.

4.3 ENSURING DATA QUALITY

4.3.1 Continuous Process Improvement

Robust data collection is critical to the success of the CIBMTR. The CPI program ensures timeliness and completeness of data forms submissions (Appendix H).

Recipient Forms

Transplant centers receive CPI reports 3 times per year (January, May, and September), listing the number of follow-up forms that were due in the previous trimester and the number and percentage of each submitted within the trimester. A form is not officially submitted until all errors are resolved and all applicable information is submitted and approved. To be compliant, centers must submit ≥90% of forms due for the trimester, for all unrelated donor transplants and for related donor and autologous transplants that have occurred since December 3, 2007.

Donor Forms

The Donor Data Management Team oversees submission of transplant donor forms from NMDP/Be The Match donor, collection, and apheresis centers. Donor CPI reports are generated 4 times per year (January, April, July, and October). To be compliant, centers must submit 100% of the forms required for that CPI period.


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4.3.2 Verification and Validation

FormsNet

When data are entered into FormsNet, a series of entry level validation checks takes place to ensure data consistency. This process flags certain errors at the time of entry and allows the CIBMTR to contact the center data manager so errors can be corrected immediately while source documents are readily available. If a data field does not pass the FormsNet validation checks, an error comment is generated, and the data manager is navigated to an error review page to review, resolve, or override the unresolved errors. Lastly, an error report is generated that lists any unresolved errors as well as errors that have been overridden.

Entry Level Validation Checks

Mandatory field validation

Range validation

Cross form consistency

Within form consistency

Core field validations

Research Database

Data extracted from FormsNet and loaded to the Research Database each month undergoes comprehensive validation and verification. These data are rigorously validated for consistency, completeness, and uniqueness using business rules implemented in custom logic for the categories provided below. Finally, the Data Quality Team reviews errors and works with transplant centers to correct data.

Extraction Validation Checks

Fields that cannot be null

Cross form consistency

Longitudinal consistency

Logical relationships

Rules across these categories were updated and tested as part of the recent Forms Revision. The CIBMTR continues to work toward reducing the number of data entry errors. Currently, the rate of form rejection due to inconsistently reported data is <2%, which is attributed to recent enhancements in transplant center education as well as to enhanced validations built in at the point of entry in FormsNet.


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4.3.3 On-Site Data Audit Program

On-going data audits are performed at all CIBMTR participating transplant centers. The audit compares data in source documents maintained at the transplant center with data contained in the CIBMTR Research Database. Clinical Research Associates perform the on-site transplant center audits, spending 3-4 days at each center reviewing original source documents. The overall audit process is displayed in Figure 4.2.

The Foundation for the Accreditation of Cellular Therapy (FACT) and the CIBMTR agreed in 2015 to consolidate transplant center data audits. Therefore, beginning in 2016, FACT auditors will use the results of the CIBMTR data audit and discontinue use of the current independent FACT data audit. Consolidating audits of transplant center data

collection and reporting eases the reporting and compliance burdens of transplant centers and demonstrates the value of the CIBMTR’s data collection and management to an international accrediting agency.

In 2015, 62 centers were scheduled for audit (48 domestic, 16 international). As of December 31, 57 centers were notified of their final audit results, including requested corrective action follow-up. Of the centers sent reports, 80% passed with ≤3% critical field errors. Of the 11 centers that did not pass the audit, 7 completed all required corrective action; the remaining 4 centers are in the process of completing requested corrective action.

Figure 4.2. Audit Process


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4.4 PROTECTING PATIENTS AND DATA

4.4.1 Human Subjects / HIPAA Compliance

The CIBMTR is committed to the ethical conduct of research. All Coordinating Center personnel maintain Collaborative IRB Training Initiative (CITI) certification. The NMDP/Be The Match IRB, which is fully accredited by the Association for the Accreditation of Human Research Protection Programs, reviews all human subject research conducted by the CIBMTR. The CIBMTR maintains compliance with the Health Insurance Portability and Accountability Act (HIPAA) Privacy Rule, as applicable. CIBMTR rules requiring the registration of all consecutive HCT recipients ensure the inclusion of women, minorities, and children, so the Research Database population includes women and minorities in the same proportion as found in the general HCT population. Children are included in most CIBMTR studies; their inclusion is dependent on the study focus.

4.4.2 Information Security and Data Privacy

The CIBMTR protects the data and information received from transplant centers and patients. The SCTOD contract requires specific protections through minimum security controls, policies, and standards. The CIBMTR’s data systems are maintained in accordance with the Federal Information Systems Management Act of 2002, with information security guidance provided by the National Institute of Standards and Technology. In accordance with National Institute of Standards and Technology Special Publication 800-18, and supervised by HRSA’s Office of Information Technology, the CIBMTR maintains a System Security Plan that outlines management, operational, and technical controls.

Since 2008, the CIBMTR holds an Authority to Operate from the Chief Information Officer of HRSA. The certification was renewed in June 2015, and security audits are performed annually, most recently in September 2015. The NMDP/Be The Match also holds an Authority to Operate from HRSA, ensuring similar standards of information security are applied to all CIBMTR and NMDP/Be The Match systems. These controls, maintained by the CIBMTR and NMDP/Be The Match, protect the data and information in these systems in ways beyond those required by HIPAA.

http://csrc.nist.gov/publications/PubsSPs.html



CIBMTR 2015 Annual Report 5.0 WHAT WE WILL DO NEXT

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5.0 WHAT WE WILL DO NEXT

In 2016, the CIBMTR will continue to conduct high quality research through a collaborative process.

Specific plans are listed in Figure 5.1 and Tables 5.2-5.4.

Figure 5.1. Plans for 2016

Details provided in Tables 5.2-5.4.

Enhance the quality and scope of CIBMTR data

Increase research

productivity and scientific

impact

Expand data and knowledge

sharing


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Table 5.2. Plans to Enhance Data

Enhance the quality and scope of CIBMTR data

Further develop a cellular therapy registry

Expand data collection to patients who receive non-HCT treatments

Continually review data collection forms for scientific content updates

Implement key data validations in FormsNet, close to the point of data capture

Implement consolidated transplant center data audits with FACT to enhance data monitoring while decreasing reporting and compliance burdens on transplant centers

Work toward an Integrated Data Warehouse to strengthen flexibility of data use and ease of data access

Provide onsite and online data management training support for transplant centers

Table 5.3. Plans to Expand Knowledge Sharing

Expand data and knowledge sharing

Implement two data sharing products using Qlikview software applications to enhance transplant centers’ self-service access to their data and information

• eDBtC will provide centers with access to a wide range of TED- and CRF-level data

• Performance data tool will provide centers with comparison data from the SCTOD annual center-specific analysis of posttransplant survival in US centers

Publish easy to read summaries of key HCT publications

Strengthen collaborations with international partners to facilitate data sharing


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Table 5.4. Plans to Increase Impact

Increase research productivity and scientific impact

Focus on studies with highest scientific impact and important clinical and policy implications; limit accepted studies to a number that can be completed in a timely manner

Support Working Committee Chairs to be active leaders in their committees

Regularly share metrics as a Working Committee management resource

Expand use of outcomes data to assist in the design, implementation, and long-term follow-up of clinical trials

Develop new statistical methodologies

Support Health Services Research initiatives

CIBMTR 2015 Annual Report 2015 KEY ACCOMPLISHMENTS

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2015 KEY ACCOMPLISHMENTS

CIBMTR by the Numbers

RESEARCH DATABASE ~400 participating centers >425,000 patients ~21,000 new patients annually

PUBLICATIONS >1,000 publications since inception

76 publications in 2015 38 from Working Committees 8 from BMT CTN 4 from Health Services Research

Program 9 from Bioinformatics Research

Program 5 from Statistical Methodology

Research Program 14 from Coordinating Center (2 cross-listed between programs)

PRESENTATIONS 65 presentations in 2015 (41 oral and

24 poster) 25 abstracts (17 oral and 8 poster)

presented at the 2015 ASH Annual Meeting

17 abstracts (13 oral and 4 poster) presented at the 2015 BMT Tandem Meetings

6 abstracts (4 oral and 2 poster) presented at the 2015 EBMT Annual Meeting

17 abstracts (7 oral and 10 poster) presented at other national and international conferences

Clinical Outcomes Research Program

WORKING COMMITTEES Administered 15 committees in which 2,300 worldwide researchers participate

Collaborated with the 54 global experts in the HCT field who voluntarily chair the committees

Conducted 176 ongoing studies

Reviewed 193 new study proposals for presentation at the 2016 BMT Tandem Meetings, >75% of which were submitted by new investigators

Presented 38 abstracts (26 oral and 12 poster) at national and international conferences

Supported approximately 400 scientific authors at 200 institutions worldwide to publish research findings

Published 38 manuscripts in peer-reviewed journals

CELLULAR THERAPIES AND NON-TRANSPLANT THERAPY

Initiated a Cellular Therapies Task Force charged with determining the type and process for cellular therapies data collection

Hosted a Cellular Therapies Forum to develop recommendations for a cellular therapy registry


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Immunobiology Research Program

Collected samples from 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks)

Curated 12,656 samples in the Research Repository (137,464 overall)

Completed high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient pairs

Distributed 8,770 samples to investigators for various studies



RESEARCH REPOSITORY Added 4,392 unrelated recipient samples (57,018 overall)

Added 1,264 related recipient samples (5,377 overall)

Added 4,819 adult unrelated donor samples (59,375 overall)

Added 1,258 related donor samples (5,128 overall)

Added 923 unrelated cord blood samples (10,566 overall)

Clinical Trials Support Program

BMT CTN Opened 3 new trials to accrual, bringing the total number of launched trials to 37

Accrued >1,100 patients to trials, increasing the total number of accrued patients to >8,500

Managed 12 open protocols with overall accrual for open studies at about 125% of projections



RCI BMT Opened 3 new trials to accrual, bringing the total number of launched trials to 15

Accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000

Presented 4 oral abstracts at national and international conferences

Survey Research Group Supported 8 active studies, and participated in the development of one upcoming study


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Health Services Research Program

Established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings

Opened a multi-center randomized controlled study to evaluate the effectiveness of an individualized survivorship care plan template; 17 centers will participate in this study

Conducted a national survey of community hematologists / oncologists on referral practices, perceptions of HCT, and education preferences to inform an educational intervention on timing of community-based referral for HCT

Completed two studies: Identifying HCT patient housing and caregiver challenges and potential interventions and The financial impact of allogeneic HCT on patient and family: a pilot study

Conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs

Provided technical plain language writing consultation; developed 3 easy-to-read consent forms and 6 patient information sheets for BMT CTN protocols

Presented 3 oral abstracts at a national conference


Bioinformatics Research Program

Developed guidelines for reporting HLA and KIR genotyping via Next Generation Sequencing

Investigated the role of genetic ancestry in transplantation

Developed methods for HLA association studies

Investigated HLA data from other countries



Statistical Methodology Research Program

Developed new statistical models

Ensured the statistical integrity of CIBMTR scientific activities

Contributed to results in articles on HCT-related statistical issues for clinical audiences

Supported Working Committee study investigators in developing scientific study protocols using CIBMTR data

Published 4 peer-reviewed manuscripts and 1 editorial


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SCTOD

Published annual Center-Specific Survival Report, Transplant Center Volumes Data for 2009-2013, and a Transplant Activity Report for transplants performed 2009-2013

Began implementing recommended changes to tools to enhance transplant centers’ quality improvement efforts in response to recommendations from the 2014 Center Outcomes Forum

Continued analysis of a quality of life project, which is unique in that the CIBMTR collected data directly from patients after HCT

In conjunction with Be The Match’s Patient and Health Professional Services, published 12 lay summaries of CIBMTR publications

CIBMTR 2015 Annual Report APPENDIX A: TRANSPLANT CENTERS

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APPENDIX A: TRANSPLANT CENTERS

Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR

CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS

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APPENDIX A1: US CENTERS

The following table lists the US-based transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.

Participating Center City State MUD RELATED AUTO

University of Alabama Birmingham Birmingham AL CRF CRF CRF

Providence Hospital HPC Transplant Center Mobile AL N/A N/A CRF

University of Arkansas for Medical Sciences Little Rock AR CRF TED N/A

Banner MD Anderson Cancer Center Gilbert AZ CRF CRF CRF

Banner Blood and Marrow Transplant Program Phoenix AZ CRF CRF CRF

Mayo Clinic Arizona and Phoenix Children's Hospital Phoenix AZ CRF CRF CRF

Cancer Transplant Institute at Virginia G. Piper Cancer Center Scottsdale AZ CRF CRF CRF

University Medical Center - Tucson Tucson AZ CRF TED TED

City of Hope National Medical Center Duarte CA CRF TED TED

Scripps Blood & Marrow Transplant Program La Jolla CA CRF CRF CRF

University of California, San Diego Medical Center La Jolla CA CRF CRF CRF

Loma Linda University Cancer Center Loma Linda CA CRF CRF CRF

Cedars Sinai Medical Center Los Angeles CA CRF TED N/A

Children's Hospital of Los Angeles Los Angeles CA CRF TED TED

UCLA Center for Health Sciences Los Angeles CA CRF CRF TED

USC BMT Program Los Angeles CA CRF CRF CRF

Children's Hospital of Oakland Oakland CA CRF CRF CRF

Children's Hospital of Orange County Orange CA CRF CRF CRF


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St Joseph’s Hospital Irvine Orange CA N/A N/A CRF

University of California Irvine Medical Center Orange CA CRF CRF CRF

Lucile Packard Children’s Hospital Palo Alto CA CRF CRF CRF

Sutter Cancer Center Sacramento CA TED TED TED

University of California - Davis Cancer Center Sacramento CA CRF TED TED

Rady Children's Hospital San Diego San Diego CA CRF CRF CRF

University of California - San Francisco - Adults San Francisco CA CRF CRF CRF

University of California - San Francisco - Pediatrics San Francisco CA CRF CRF CRF

Stanford Health Care Stanford CA CRF CRF TED

Stanford University Medical Center Stanford CA CRF CRF TED

The Children's Hospital of Denver Aurora CO CRF CRF CRF

University of Colorado Hospital Aurora CO CRF TED TED

Colorado Blood Cancer Institute Denver CO CRF TED TED

University of Colorado Medical School Denver CO N/A N/A N/A

University of Connecticut Farmington CT N/A N/A N/A

Yale New Haven Hospital New Haven CT CRF CRF CRF

Children's National Medical Center Washington DC CRF TED TED

Medstar Georgetown University Hospital Washington DC CRF N/A CRF

Christiana Care Newark DE CRF CRF CRF

Alfred I. duPont Hospital for Children Wilmington DE CRF CRF CRF

Shands HealthCare & University of Florida Gainesville FL CRF CRF CRF


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BMT Program of Mayo Clinic / Nemours and Wolfson Children's Hospital Jacksonville FL CRF CRF CRF

Mayo Clinic Florida - Jacksonville Jacksonville FL CRF CRF CRF

Miami Children's Hospital Miami FL CRF TED TED

University of Miami - Adults Miami FL CRF CRF CRF

University of Miami / Jackson Memorial Hospital Miami FL CRF TED TED

Blood & Marrow Transplant Center, Florida Hospital Medical Group Orlando FL CRF CRF CRF

Memorial Cancer Institute Pembroke Pines FL TED TED TED

All Children's Hospital St. Petersburg FL CRF CRF CRF

H Lee Moffitt Cancer Center Tampa FL CRF TED TED

Children's Healthcare of Atlanta at Egleston Atlanta GA CRF CRF CRF

Emory University Atlanta GA CRF CRF CRF

The Blood and Marrow Transplant Program at Northside Hospital Atlanta GA CRF CRF CRF

Georgia Regents University Cancer Center Augusta GA CRF CRF CRF

Hawaii Medical Center Honolulu HI N/A N/A N/A

Kapi'olani Medical Center for Women and Children Honolulu HI CRF CRF CRF

University of Iowa Hospital & Clinics Iowa City IA CRF CRF CRF

St. Luke's Mountain States Tumor Institute Boise ID N/A N/A CRF

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago IL CRF CRF CRF

Northwestern Memorial Hospital Chicago IL CRF CRF TED

Northwestern Memorial Hospital - Department of Immunotherapy Chicago IL CRF TED N/A

The Coleman Foundation Blood and Marrow Transplant Center, Rush University Chicago IL CRF CRF CRF


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University of Chicago Medical Center Chicago IL CRF CRF CRF

University of Illinois at Chicago Medical Center Chicago IL CRF CRF CRF

Loyola University Medical Center Maywood IL CRF CRF CRF

Advocate Lutheran General Hospital Park Ridge IL N/A TED TED

Cancer Treatment Centers of America - Midwest Zion IL CRF CRF CRF

Indiana Blood & Marrow Transplantation Beech Grove IN CRF CRF CRF

Indiana University Hospital / Riley Hospital for Children Indianapolis IN CRF CRF TED

St Vincent Hospital Indianapolis Indianapolis IN N/A CRF CRF

University of Kansas Kansas City KS CRF CRF CRF

Via Christi Hospitals Wichita Wichita KS N/A CRF CRF

University of Kentucky Medical Center Lexington KY CRF TED TED

University of Louisville Hospital / James Brown Cancer Center Louisville KY CRF TED TED

Louisiana State University Children's Hospital New Orleans LA CRF CRF CRF

Ochsner Medical Center New Orleans LA N/A CRF TED

Tulane University Medical Center New Orleans LA CRF CRF CRF

Louisiana State University Health Sciences Center - Shreveport Shreveport LA CRF CRF CRF

Beth Israel Deaconess Medical Center Boston MA CRF TED TED

Boston Medical Center Boston MA N/A N/A TED

Dana Farber Cancer Institute at Brigham and Women's Hospital - Adults Boston MA CRF CRF TED

Dana Farber Cancer Institute at Brigham and Women's Hospital - Pediatrics Boston MA CRF TED TED

Massachusetts General Hospital Boston MA CRF TED TED


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Tufts New England Medical Center Boston MA CRF TED TED

Lahey Clinic Medical Center Burlington MA N/A N/A TED

UMass Memorial Medical Center Worcester MA CRF CRF CRF

Johns Hopkins Oncology Center Baltimore MD TED TED TED

University of Maryland School of Medicine Baltimore MD CRF CRF CRF

National Heart Lung & Blood Institute Bethesda MD N/A TED N/A

National Institutes of Allergy & Infectious Disease Bethesda MD N/A TED N/A

National Institutes of Health Bethesda MD N/A TED N/A

NIH - NCI Experimental Transplantation and Immunology Branch (Related Donor Program)

Bethesda MD N/A CRF CRF

NIH - NCI Matched Unrelated Donor Program Bethesda MD CRF N/A N/A

The University of Michigan Ann Arbor MI CRF TED TED

Children's Hospital of Michigan Detroit MI CRF CRF CRF

Henry Ford Hospital Bone Marrow Transplant Program Detroit MI CRF CRF CRF

Karmanos Cancer Institute Detroit MI CRF CRF CRF

Helen DeVos Children's Hospital Grand Rapids MI CRF CRF CRF

Spectrum Health Grand Rapids MI CRF CRF CRF

Abbott Northwest Hospital Minneapolis MN N/A N/A TED

Masonic Cancer Center University of Minnesota Minneapolis MN CRF CRF CRF

Mayo Clinic Rochester Rochester MN CRF CRF CRF

Saint Luke’s Blood & Marrow Transplant Program Kansas City MO CRF CRF CRF


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The Children's Mercy Hospitals and Clinics Kansas City MO CRF CRF CRF

Cardinal Glennon Children's Hospital St. Louis MO CRF CRF CRF

SSM Health Saint Louis University Hospital St. Louis MO CRF TED TED

Washington University School of Medicine St. Louis MO CRF CRF TED

Washington University / St Louis Children's Hospital St. Louis MO CRF CRF CRF

University of Mississippi Medical Center - Jackson Jackson MS CRF TED TED

Billings Clinic Cancer Center Billings MT N/A N/A TED

University of North Carolina Hospitals - Chapel Hill Chapel Hill NC CRF CRF CRF

BMT Program at Levine Children's Hospital / Carolinas Medical Center Charlotte NC CRF CRF CRF

Levine Cancer Institute Charlotte NC N/A CRF CRF

Duke University - Adults Durham NC CRF TED TED

Duke University Medical Center, Pediatric BMT Durham NC CRF CRF CRF

Duke University, Immunology / BMT, Pediatrics Durham NC N/A TED N/A

Novant Health Oncology Specialists Winston-Salem NC N/A N/A N/A

Wake Forest Baptist Health Winston-Salem NC CRF TED TED

CHI Health Bergan Mercy Omaha NE N/A N/A TED

CHI Health Immanuel Omaha NE N/A N/A TED

Nebraska Methodist Hospital Omaha NE N/A N/A TED

University of Nebraska Medical Center Omaha NE CRF CRF CRF

Dartmouth-Hitchcock Medical Center Lebanon NH CRF CRF CRF

Hackensack University Medical Center Hackensack NJ CRF CRF CRF


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Cancer Institute of New Jersey New Brunswick NJ CRF CRF CRF

University of New Mexico - Pediatrics Albuquerque NM N/A N/A CRF

Childrens Hospital at Montefiore Bronx NY CRF TED TED

Montefiore Medical Center Bronx NY CRF CRF TED

Roswell Park Cancer Institute Buffalo NY CRF CRF CRF

Westchester Medical Center Hawthorne NY CRF CRF CRF

North Shore University Hospital Manhasset NY CRF TED TED

Cohen Children's Medical Center of New York

New Hyde Park NY CRF CRF CRF

Memorial Sloan Kettering Cancer Center - Adults New York NY CRF TED TED

Memorial Sloan Kettering Cancer Center - Pediatrics New York NY CRF TED TED

Morgan Stanley Children's Hospital of New York New York NY CRF CRF CRF

Mount Sinai Medical Center - New York New York NY CRF CRF TED

New York Presbyterian Hospital New York NY CRF CRF CRF

New York Presbyterian Hospital / Columbia University Medical Center New York NY CRF CRF CRF

New York University Medical Center New York NY TED TED TED

St Vincent's Hospital Manhattan New York NY N/A TED TED

University of Rochester Medical Center Rochester NY CRF CRF CRF

Stony Brook University Medical Center Stony Brook NY TED TED CRF

State University of NY Upstate Medical University Syracuse NY N/A CRF CRF

Akron Children's Hospital Akron OH CRF CRF CRF

Cincinnati Children's Hospital Cincinnati OH CRF CRF CRF


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Jewish Hospital Blood and Marrow Transplant Center Cincinnati OH CRF CRF CRF

University of Cincinnati Medical Center Cincinnati OH CRF CRF CRF

Cleveland Clinic Cleveland OH CRF CRF CRF

Seidman Cancer Center - University Hospitals Case Medical Center Cleveland OH CRF CRF CRF

Nationwide Children's Hospital Columbus OH CRF CRF CRF

The Ohio State University Medical Center Columbus OH CRF CRF CRF

Miami Valley Hospital BMT Program Dayton OH N/A N/A CRF

Oklahoma University Medical Center Oklahoma City OK CRF CRF CRF

Cancer Treatment Centers of America Tulsa OK CRF CRF CRF

Saint Francis Hospital - Oklahoma Tulsa OK N/A TED TED

Legacy Good Samaritan Hospital and Medical Center Portland OR N/A N/A CRF

Oregon Health and Science University Portland OR CRF CRF CRF

Pediatric BMT Program, Doernbecher Children's Hospital (OHSU) Portland OR CRF CRF CRF

Providence Portland Medical Center Portland OR N/A N/A CRF

Geisinger Medical Center Danville PA CRF TED TED

Penn State Hershey Medical Center Hershey PA CRF CRF CRF

Abramson Cancer Center University - Pennsylvania Medical Center Philadelphia PA CRF CRF CRF

Eastern Regional Medical Center Philadelphia PA N/A CRF CRF

Hahnemann University Hospitals Philadelphia PA CRF TED TED

Philadelphia Children's Hospital Philadelphia PA TED TED N/A

St Christopher's Hospital for Children Philadelphia PA TED TED TED


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Temple Bone Marrow Transplant Program Philadelphia PA CRF TED TED

Thomas Jefferson University Philadelphia PA TED TED TED

Children's Hospital of Pittsburgh of UPMC Pittsburgh PA CRF CRF CRF

University of Pittsburgh Medical Center Pittsburgh PA CRF CRF CRF

West Penn Hospital Pittsburgh PA CRF CRF CRF

Roger Williams Medical Center Providence RI CRF TED TED

Charleston Hematology Oncology Charleston SC N/A CRF CRF

Medical University of South Carolina Charleston SC CRF TED TED

Richland Memorial Hospital Columbia SC N/A N/A N/A

Cancer Centers of the Carolinas Greenville SC CRF CRF CRF

Saint Francis Hospital - Greenville Greenville SC N/A CRF CRF

Avera Mckennan Transplant Institute Sioux Falls SD CRF CRF CRF

Thompson Cancer Survival Center Knoxville TN N/A N/A TED

Baptist Blood and Marrow Transplant Memphis TN CRF CRF CRF

Baptist Centers for Cancer Care Memphis TN TED TED TED

St Jude Children's Research Hospital Memphis TN CRF TED N/A

The West Clinic / Methodist Healthcare Blood and Marrow Transplant Center Memphis TN N/A N/A TED

University of Tennessee Memphis TN CRF CRF CRF

Sarah Cannon BMT Center at Centennial Medical Center Nashville TN CRF TED TED

Vanderbilt University Nashville TN CRF TED TED

Vanderbilt University Veterans Center Nashville TN TED TED N/A


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Texas Oncology Amarillo TX CRF TED TED

Southwest Regional Cancer Center Austin TX N/A N/A N/A

Baylor University Medical Center Dallas TX CRF CRF CRF

Children's Medical Center - Dallas Dallas TX CRF CRF CRF

Medical City Dallas Hospital Dallas TX CRF CRF CRF

UT Southwestern Medical Center - BMT Program Dallas TX CRF CRF CRF

Cook Children's Medical Center Fort Worth TX CRF CRF CRF

Baylor College of Medicine Houston TX CRF CRF CRF

MD Anderson Cancer Center Houston TX CRF CRF N/A

Wilford Hall Medical Center Lackland AFB TX TED TED TED

Covenant Health System Hematopoietic Transplant Program Lubbock TX N/A TED TED

Texas Tech University Medical Center Lubbock TX CRF CRF CRF

South Texas Veterans Health Care System San Antonio TX N/A CRF CRF

Texas Transplant Institute San Antonio TX CRF CRF CRF

University of Texas Health Science Center San Antonio TX N/A CRF CRF

Scott and White Memorial Hospital Temple TX N/A N/A CRF

Latter Day Saints Hospital Salt Lake City UT CRF CRF CRF

University of Utah Medical Center Salt Lake City UT CRF CRF CRF

Utah Blood and Marrow Transplant Program - Adults Salt Lake City UT CRF CRF CRF

Utah Blood and Marrow Transplant Program - Pediatrics Salt Lake City UT CRF CRF CRF

University of Virginia Health System Charlottesville VA CRF CRF CRF


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Fairfax - Northern Virginia Hospital Fairfax VA CRF CRF CRF

Virginia Oncology Associates Norfolk VA N/A N/A TED

Virginia Commonwealth University Massey Cancer Center BMT Program Richmond VA CRF CRF CRF

University of Vermont Cancer Center Burlington VT N/A N/A TED

Fred Hutchinson Cancer Center Seattle WA CRF CRF CRF

VA Puget Sound Healthcare System Seattle WA TED TED N/A

University of Wisconsin Hospital and Clinics Madison WI CRF CRF CRF

Marshfield Clinic Marshfield WI N/A N/A CRF

Aurora St Luke's Medical Center Milwaukee WI N/A N/A CRF

Children's Hospital of Wisconsin Milwaukee WI CRF CRF CRF

Froedtert & Medical College of Wisconsin Milwaukee WI CRF CRF TED

Osborn Hematopoietic Malignancy & Transplantation Program Morgantown WV CRF CRF CRF

CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS

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APPENDIX A2: INTERNATIONAL CENTERS

The following table lists the international transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF.

Participating Center City Country MUD RELATED AUTO

Fundaleu-Angelica Ocampo Buenos Aires Argentina CRF CRF CRF

Hospital Universitario Austral Buenos Aires Argentina CRF CRF CRF

Hospital Privado de Cordoba Cordoba Argentina CRF CRF CRF

Royal Adelaide Hospital / SA Pathology Adelaide Australia CRF CRF N/A

Royal Prince Alfred Hospital Camperdown Australia CRF CRF TED

St Vincent's Hospital Darlinghurst Australia TED TED N/A

Royal Brisbane & Women's Hospital Herston Australia CRF CRF CRF

Alfred Hospital Melbourne Australia TED TED TED

Royal Children's Hospital Parkville Australia CRF CRF N/A

Fiona Stanley Hospital Perth Australia CRF CRF N/A

Princess Margaret Hospital for Children Perth Australia CRF CRF TED

Royal Perth Hospital Perth Australia CRF CRF N/A

Sydney Children's Hospital Randwick Australia CRF CRF N/A

Lady Cilento Children’s Hospital South Brisbane Australia N/A CRF CRF

Royal Melbourne Hospital Victoria Australia CRF CRF N/A

Calvary Mater Newcastle Hospital Waratah Australia N/A N/A CRF

Children's Hospital at Westmead Westmead Australia CRF CRF TED

Westmead Hospital Westmead Australia CRF CRF N/A


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St Anna Children's Hospital Vienna Austria TED TED N/A

Children's University Hospital Bruxelles Belgium TED TED TED

University Hospital Antwerp Edegem Belgium TED TED N/A

University Hospital Gasthuisberg Leuven Belgium TED TED N/A

University Estadual de Campinas Campinas Brazil CRF CRF CRF

Hospital de Clinicas Curitiba Curitiba Brazil CRF CRF CRF

Hospital Amaral Carvalho Jau Brazil CRF CRF CRF

Hospital de Porto Alegre Porto Alegre Brazil TED TED TED

Instituto Nacional de Cancer Rio de Janeiro Brazil CRF CRF CRF

Albert Einstein Hospital Sao Paolo Brazil TED TED TED

Hospital Sirio Libanes Sao Paolo Brazil CRF CRF CRF

Instituto de Oncologia Pediatrica Sao Paolo Brazil TED TED TED

Alberta Children's Hospital Calgary Canada CRF CRF CRF

Tom Baker Cancer Centre Calgary Canada TED TED TED

Queen Elizabeth II Health Sciences Center Halifax Canada TED TED TED

Hamilton Health Sciences Hamilton Canada CRF CRF CRF

Kingston General Hospital Kingston Canada N/A N/A TED

Centre Hospitalier Montreal Canada CRF CRF CRF

Maisonneuve - Rosemont Hospital Montreal Canada TED TED N/A

McGill University Health Centre - Royal Victoria Hospital Montreal Canada TED TED N/A

Montreal Children's Hospital Montreal Canada TED TED TED


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Hotel-Dieu de Quebec Quebec Canada TED TED TED

CHA-Enfant-Jesus Hospital Quebec City Canada TED TED TED

Saint John Regional Hospital Saint John Canada N/A N/A CRF

St John's Health Sciences Center St. John's Canada N/A N/A TED

Health Sciences North Sudbury Canada N/A N/A TED

Princess Margaret Hospital (alloHCT) Toronto Canada CRF CRF N/A

Princess Margaret Hospital (autoHCT) Toronto Canada N/A N/A CRF

British Columbia Children's Hospital Vancouver Canada CRF CRF CRF

Vancouver General Hospital Vancouver Canada N/A N/A N/A

CancerCare Manitoba / University of Manitoba Winnipeg Canada CRF CRF CRF

Instituto de Trasplante de Medula Osea de la Costa Caribe Barranquilla Colombia TED TED TED

Clinica de Marly Bogota Colombia CRF CRF CRF

Charles University Hospital - Pilsen Pilsen Czech Republic CRF CRF CRF

Institute of Hem-Blood Transfusion Praha Czech Republic TED TED N/A

University Hospital, Rigshospitalet Copenhagen Denmark CRF CRF N/A

Children's Cancer Hospital - Egypt Cairo Egypt N/A TED TED

NCI Cairo University Cairo Egypt TED TED N/A

Helsinki University Central Hospital Helsinki Finland CRF CRF N/A

Turku University Turku Finland TED TED N/A

Centre Hospitalier Regional University D'Angers Angers France TED TED N/A

Hopital Jean Minjoz Besançon France CRF CRF N/A


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Hopital Claude Huriez, Lille Lille France MED-A MED-A N/A

Hospital Edouard Herriot Lyon France MED-A MED-A N/A

Institute Paoli Calmettes Marseille France CRF CRF N/A

Hospital Saint Louis Paris France CRF CRF N/A

Hospital Jean Bernard Poitiers France CRF CRF N/A

University Hospital of Essen Essen Germany CRF CRF CRF

University Children’s Hospital Frankfurt Frankfurt Germany CRF CRF CRF

Albert-Ludwig University - Freiburg Freiburg Germany CRF CRF N/A

UKE Hamburg, Klinik und Poliklinik für Stammzelltransplantation Hamburg Germany CRF CRF N/A

University of Heidelberg Heidelberg Germany CRF CRF N/A

Christian Albrechts University Kiel Germany CRF CRF CRF

University Leipzig, BMT Center Leipzig Germany CRF N/A N/A

University Hospital Mainz Mainz Germany TED TED TED

University of Munich Munich Germany CRF CRF N/A

Klinikum der Universitӓt Regensburg Regensburg Germany MED-A MED-A N/A

Universitӓts Klinikum Tubingen Tübingen Germany CRF TED N/A

Universitӓts Kinderklinik Tubingen Tübingen Germany CRF TED N/A

Universitӓt Ulm - Adults Ulm Germany CRF CRF TED

Deutsche Klinik für Diagnostik - Wiesbaden Wiesbaden Germany CRF CRF N/A

University Hospital of Patras, Patras University Medical Center Rio Patras Greece TED TED TED

Chinese University of Hong Kong Shatin Hong Kong CRF CRF N/A


Page | 69


Gujrat Cancer & Research Institute Ahmedabad India CRF CRF CRF

Sir Ganga Ram Hospital Delhi India CRF CRF CRF

Apollo Hospital International Ltd Gandhinagar India N/A TED TED

Fortis Memorial Research Institute Gurgaon India N/A CRF CRF

Bone Marrow Transplant Unit, Christian Medical College, Ludhiana Ludhiana India TED TED TED

Rajiv Gandhi Cancer Institute and Research Centre New Delhi India CRF CRF CRF

Jawaharlal Institute of Postgraduate Medical Education and Research Puducherry India N/A TED TED

Sahyadri Speciality Hospital Pune India CRF CRF CRF

Christian Medical College Hospital Vellore India CRF CRF N/A

Rambam Medical Center Haifa Israel CRF CRF CRF

Haddasah University Hospital Jerusalem Israel CRF CRF N/A

Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital Petah Tikva Israel CRF CRF CRF

Tel-Aviv Sourasky Medical Center Tel-Aviv Israel CRF CRF CRF

Chaim Sheba Medical Center - Pediatrics

Tel-Hashomer Israel CRF CRF CRF

Sheba Medical Center Tel-Hashomer Israel CRF CRF N/A

Instituto di Ematologia e Oncologia Medica Seragnoli Bologna Italy MED-A MED-A N/A

University Bologna - Pediatrics Bologna Italy TED TED TED

St Eugenio Hospital Rome Italy CRF CRF N/A

Ospedale Molinette Torino Italy MED-A MED-A N/A

University Torino Torino Italy TED TED N/A

Osaka City University Osaka Japan CRF CRF N/A


Page | 70


Hospital Universitario Monterrey Mexico CRF CRF CRF

Centro de Hematologia y Medicina Interna Clinica RUIZ de Puebla Puebla Mexico TED TED TED

Academic Medical Center Amsterdam Netherlands CRF TED TED

VU Medical Center - Amsterdam Amsterdam Netherlands CRF TED TED

University Medical Center Groningen Groningen Netherlands CRF N/A N/A

Leiden University Medical Center Leiden Netherlands CRF TED N/A

Academic Hospital Maastricht Maastricht Netherlands CRF TED N/A

University Hospital of Nijmegen Nijmegen Netherlands CRF TED N/A

Erasmus MC - Daniel den Hoed Cancer Center Rotterdam Netherlands CRF TED N/A

University Hospital Utrecht Utrecht Netherlands CRF N/A N/A

University Medical Center Utrecht - Pediatrics Utrecht Netherlands CRF N/A N/A

Auckland City Hospital Auckland New Zealand CRF CRF N/A

Starship Children's Hospital Auckland New Zealand CRF CRF N/A

Christchurch Hospital Christchurch New Zealand CRF CRF TED

Wellington Blood and Cancer Centre Wellington New Zealand CRF CRF N/A

Rikshospitalet - The National Hospital Oslo Norway CRF N/A N/A

Hill Park Hospital - National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi Pakistan CRF CRF CRF

Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan N/A CRF CRF

Instituto Nacional Salud Del Niño San Borja San Borja Peru N/A TED N/A

Silesian Medical Academy Katowice Poland CRF CRF N/A


Page | 71


Poznan University of Medical Sciences Poznan Poland MED-A MED-A N/A

Lower-Silesian Center for Cellular Transplantation and National Bone Marrow Donor Registry

Wroclaw Poland CRF CRF N/A

Institute Portugues de Oncologia - Lisbon Lisbon Portugal CRF CRF N/A

Institute Portugues de Oncologia - Porto Porto Portugal N/A N/A N/A

St Petersburg State Medical University St. Petersburg

Russian Federation TED TED TED

King Faisal Specialist Hospital - Pediatrics Riyadh Saudi Arabia CRF CRF N/A

King Faisal Specialist Hospital & Research Center - Adults Riyadh Saudi Arabia CRF CRF N/A

National University Health System - Adults Singapore Singapore CRF CRF CRF

National University Health System - Pediatrics Singapore Singapore CRF CRF CRF

Parkway Cancer Centre Singapore Singapore CRF CRF CRF

Singapore General Hospital Singapore Singapore CRF CRF N/A

Slovak Medical University Bratislava Slovak Republic TED TED N/A

Constantiaberg Medi-Clinic Cape Town South Africa CRF CRF CRF

University of Witwatersrand Parktown South Africa CRF CRF CRF

Asan Medical Center Seoul South Korea CRF CRF N/A

Samsung Medical Center Seoul South Korea CRF CRF CRF

Seoul St. Mary's Hospital Catholic BMT Center Seoul South Korea CRF CRF CRF

University Barcelona Barcelona Spain TED TED N/A

Gregorio Maranon University General Hospital Madrid Spain CRF CRF CRF

Hospital Puerta Hierro Madrid Spain MED-A MED-A N/A


Page | 72


Hospital Universitario Marques de Valdecilla Santander Spain TED TED TED

Hospital Universitario La Fe Valencia Spain CRF CRF N/A

Sahlgrenska University Hospital Gothenborg Sweden CRF CRF N/A

University Hospital of Lund Lund Sweden MED-A MED-A N/A

Karolinska University Hospital, Centre for Allogeneic Stem Cell Transplantation

Stockholm Sweden CRF CRF N/A

University Hospital - Uppsala Uppsala Sweden CRF CRF N/A

Basel Kantonsspital Basel Switzerland CRF CRF N/A

University Hospital-Zurich Zurich Switzerland MED-A MED-A N/A

Chang Gung Children's Hospital Taoyuan Taiwan CRF CRF CRF

King Chulalongkorn Memorial Hospital Bangkok Thailand TED TED TED

Ankara University Faculty of Medicine Ankara Turkey TED TED N/A

Gulhane Military Medical Academy Ankara Turkey CRF CRF CRF

Medical Park Hospital - Antalya Istanbul Turkey CRF CRF CRF

Birmingham Children's Hospital Birmingham United Kingdom TED TED N/A

Queen Elizabeth Hospital - Birmingham Birmingham United

Kingdom CRF CRF N/A

Bristol Children's Hospital Bristol United Kingdom CRF CRF N/A

Addenbrooke's NHS Trust Cambridge United Kingdom CRF CRF N/A

Beatson West of Scotland Cancer Centre Glasgow United

Kingdom MED-A MED-A N/A

Royal Hospital for Sick Children Glasgow United Kingdom CRF CRF N/A

Imperial College / Hammersmith Hospital London United

Kingdom CRF CRF N/A


Page | 73


Imperial College - St Mary's Hospital London United Kingdom CRF CRF N/A

The Royal Free Hampstead NHS Trust London United Kingdom MED-A MED-A N/A

Royal Marsden Hospital Sutton United Kingdom N/A N/A N/A

British Hospital Montevideo Uruguay CRF CRF CRF

Centro de Trasplante del Servicio Medico Integral (SMI) Montevideo Uruguay CRF CRF CRF

Hospital Maciel Montevideo Uruguay CRF CRF CRF

Ciudad Hospitalaraia Dr Enrique Tejera Valencia Venezuela TED TED TED

APPENDIX B: COORDINATING CENTER CIBMTR 2015 Annual Report ORGANIZATIONAL STRUCTURE AND PERSONNEL

Page | 74

APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP

The CIBMTR Coordinating Center resides on two campuses: one is located at MCW in Milwaukee, WI, and the other is located at NMDP/Be The Match in Minneapolis, MN. The Coordinating Center provides administrative, statistical, data management, clinical trials, IT, and personnel support for CIBMTR activities, and it benefits from a unique, collegial partnership with the Division of Biostatistics of MCW.

CIBMTR Milwaukee has approximately 70 employees and is an academic division of the MCW Department of Medicine. The Milwaukee office receives administrative support from the MCW departments of Grants and Contracts, Development, Office of Technology Development, Public Affairs, Human Resources and the Department of Medicine Administration. CIBMTR Minneapolis has approximately 120 employees, and several NMDP/Be The Match departments provide support for CIBMTR activities, including Finance, Contracts, and Marketing & Communications.

CIBMTR 2015 Annual Report APPENDIX B: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS

Page | 75

APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS

Chief Scientific DirectorM Horowitz, MD, MS

AdministratorCIBMTR MilwaukeeP Steinert, PhD, MBA

Medical Faculty

Senior Research Advisor

D Weisdorf, MD1

Senior Scientific Director

L Burns, MD2

M Eapen, MD, MSS Lee, MD, MPH3

M Pasquini, MD, MSJD Rizzo, MD, MSB Shaw, MD, PhDScientific DirectorM Arora, MD,MS1

A D’Souza, MBBS, MDM Hamadani, MD

P Hari, MD, MSM Riches, MD, MS4

W Saber, MD, MSE Thiel, MD, MS

Program Manager, Business

OfficeP Vespalec

Program Coordinator II

R DunnAdministrative

Assistant, SrK Jackson

AdministrativeAssistant

DL CampbellJ Claas

Program Manager,

SCTODC Doleysh

Meetings Program Manager

D Waldoch Snyder

Program Director, Statistics & Clinical Outcomes Research

W Pérez, MPH

Senior Biostatistician

J CarrerasM ChenH Wang

BiostatisticianK Bo-Subait

A DiGilioM FeiN He

M HemmerK Hu

J HuangH Millard

A St. MartinX Zhu

Program Coordinator IM Geronime

Director, ITE Bergman, MBA, MS

Database Administrator

B LiuProgrammer

Analyst IVH Tian

ProgrammerAnalyst IIIP Gengler

J GierDatabase Analyst III

X ZhangProgrammer

Analyst IIT Graczkowski

Database Analyst IM Desai

Metadata AnalystA KummerowManager IS I (part time)

C Zhang

Program Director, Advancement

S Fisher

Medical WriterJ Gillis-Smith


M BreyM Simaytis

Communications SpecialistV Vlach

Program Director, Data Operations

J Brunner-Grady, PA-C

Manager, Data

OperationsS Meiers


K BhavsarClinical Research

Coordinator IIA Prentice

T HuntProgram

Coordinator IA Pope

Clinical Research Coordinator I

A Benoit

Study Clinical Research

CoordinatorS Kulkarni

Clinical Research

Assistant IIIC AbelClinical

Research Assistant II

M PatelData EntryA Pereles

Program/IS ManagerT Moerke

Bus Systems Analyst Assoc

TBDInformation

Security AnalystJ RineckDesktop Support

TechnicianV Buendia

Grants and Contracts Financial Manager

C Gonzalez

1University of Minnesota2NMDP/Be The Match3Fred Hutchinson Cancer Research Center4University of North Carolina

CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS

Page | 76

APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 1)

Chief Scientific DirectorMary M Horowitz, MD, MS

Associate Scientific DirectorD Confer, MD

Vice President, CIBMTR MinneapolisR King, MPH

Director, CIBMTR IT SystemsM Prestegaard

Data Solutions ManagerR Renner

Senior Metadata Analyst

W ZhangMetadata Analyst

M NychS Sorensen

Principal Data Architect

E ZinkBusiness Architect

B Burgess

Senior Manager, Program Management

K Gee

Principal Business Systems Analyst

E NarrSenior Business Systems Analyst

S EwerBusiness Systems

AnalystD Kloker

Project ManagerS FreemanK O’Connor

A PullB Wakaruk

Project CoordinatorDM Campbell

Solutions ManagerE Chan

Senior System Administrator

C YangBusiness Intelligence Solutions Architect

TBD Principal Software

EngineerC Giddegowda

Senior Software EngineerA GomezN HoodJ Smith

Software EngineerZ AhmedC JordahlG Ogega

Software Engineer ETLN AryalS Stagg

Associate System Administrator

A Westin

Quality Assurance ManagerV Yarra

Senior Data Analyst

D McDonellK Schaper

T WirthSenior Quality

Assurance AnalystB SambaQuality

Assurance Analyst

V MurukurthyJ Oakes

Director, Immunobiologyand Observational Research

S Spellman, MBS1

Senior Biostatistician

P ChitphakdiathaiM HaagensonBiostatistician

D KieferS Rouse

Principal Immunobiology

Research ScientistA Howard

C Vierra-GreenSenior

Immunobiology Research Scientist

M BrownImmunobiology

Research Scientist C Brady

D SchellerS Waldvogel

Associate Immunobiology

Research ScientistA Spahn

Project CoordinatorH Severance

AdministrativeSpecialistA Carlson

Director, Bioinformatics M Maiers, MS1

Senior Bioinformatics

ScientistsA MadboulyE Williams

Bioinformatics Scientists

S FingersonM Halagan

D Roe

1Also serves as Scientific Director

CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS

Page | 77

APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 2)

Chief Scientific DirectorMary M Horowitz, MD, MS

Associate Scientific DirectorD Confer, MD

Vice President, CIBMTR MinneapolisR King, MPH

Senior Manager, Data ManagementM Matlack

Supervisor, Quality Control

B Levesque

Quality ControlSpecialistK Kutzner

Data EntryCoordinator II

L HorneData Entry

Coordinator IP Lee

K XongImaging Assistant II

N GibitzT Winder

Imaging AssistantJ Lund

Supervisor, Clinical Research,

Donor Data Management

A Hauck

Clinical Research Coordinator III

A Ewer Clinical Research

Coordinator IIR Krunkkala

K MalumS Tasky

Supervisor, Clinical Research,

Recipient DataManagement

K Gardner

Clinical Research Coordinator III

E JohnsonClinical Research

Coordinator IIA DraxlerA Mitsch

A MussetterK NutterC OlsonT Thole

Clinical Research Coordinator I

E MitchemP Wallace

Clinical Research

Coordinator IIIJ Bloomquist

S LoganE Love

Training & Development

SpecialistL Colt

Sr Research DeveloperM Unekis

TBD

Administrative AssistantM Ammi

Senior Manager,Monitoring & Auditing

D Christianson

Senior Clinical ResearchAssociateW Affield

A BirchM Petcoff

L Wendland Clinical Research

AssociateA Hendrickson

K KaneC Newcomb

K PhalenM ProueK Reilly

Clinical ResearchSpecialist

E EichAudit Coordinator

E Nordquist

Senior Manager,Prospective Research

R Drexler

Senior Clinical Research Specialist

A AdamsS Flesch

C JohnsonH Kobusingye

Clinical Research SpecialistR Besser

C PetroskeJ Vogel

Clinical Research Assistant

TBD

Prospective Research

CoordinatorJ Dworski

Survey Research Group

SupervisorD Mattila

Research Interviewer

C GriffithC Jacox

C KunakomS Lease

TBDSurvey

Research AssistantE Michem

Senior Human Research

Protection Specialist - IRB

T RouseSenior Human

Research Protection Specialist

C JobeHuman

Research Protection Specialist

J Tkachenko

Administrative SpecialistM Young

BMT CTN Project

ManagerA Foley

CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL

Page | 78

APPENDIX B3: COORDINATING CENTER LEADERSHIP

SENIOR LEADERSHIP

Mary M. Horowitz, MD, MS • Chief Scientific Director for the CIBMTR • Principal Investigator for the Data and Coordinating Center of the

BMT CTN • Research Director for the SCTOD • Robert A. Uihlein Professor of Hematologic Research at MCW • Chief of the Division of Hematology and Oncology at MCW • Attending physician in the MCW HCT program

Jeffrey Chell, MD • Executive Director for the CIBMTR • Chief Executive Officer of NMDP/Be The Match

Dennis Confer, MD • Associate Scientific Director for CIBMTR Minneapolis • Co-PI of the Data and Coordinating Center of the BMT CTN • Co-Scientific Director of the RCI BMT • Scientific Director of the Donor Health and Safety Working Committee • Chief Medical Officer of NMDP/Be The Match • Treasurer of the Executive Board of the Worldwide Network for Blood

and Marrow Transplantation

Daniel Weisdorf, MD • Senior Research Advisor for the CIBMTR • Scientific Director of the Acute Leukemia Working Committee • Vice President of the Worldwide Network for Blood and Marrow

Transplantation • Professor of Medicine at the University of Minnesota • Chief of the Division of Hematology, Oncology, and Transplant at the

University of Minnesota • Associate Chair of Clinical Research in the Department of Medicine at

the University of Minnesota • Attending physician in the University of Minnesota HCT program

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_HorowitzMM.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_ChellJ.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_ConferD.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_WeisdorfD.pdf


Page | 79

Mei-Jie Zhang, PhD • Chief Statistical Director for the CIBMTR • Biostatistician for the Acute Leukemia and Graft Sources and

Manipulation Working Committees • Professor of Biostatistics at MCW

Mary Eapen, MBBS, MS • Senior Scientific Director of Research Operations for the CIBMTR • Scientific Director for the Graft Sources and Manipulation Working

Committee as well as the Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committee

• Protocol Officer for several BMT CTN trials • Professor of Medicine at MCW • Attending physician in the MCW HCT program

J. Douglas Rizzo, MD, MS • Senior Scientific Director and Principal Investigator of the SCTOD for

the CIBMTR • Professor of Medicine at MCW • Associate Director of Clinical Operations for the Froedtert and MCW

Cancer Center • Attending physician in the MCW HCT program

Bronwen Shaw MD, PhD • Senior Scientific Director of Data Operations for the CIBMTR • Co-Scientific Director of the RCI BMT • Scientific Director of the Late Effects and Quality of Life Working

Committee as well as the Donor Health and Safety Working Committee

• Professor of Medicine at MCW • Attending physician in the MCW HCT program

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_ZhangMJ%20%282%29.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_EapenM.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_RizzoJD.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_ShawB.pdf


Page | 80

Linda Burns, MD • Senior Scientific Director of the Health Services Research Program for

the CIBMTR • Co-Scientific Director of the RCI BMT • Vice President and Medical Director of NMDP/Be The Match • Immediate Past President of the American Society of Hematology

Marcelo Pasquini, MD, MS • Senior Scientific Director of CIBMTR Clinical Trials Support – BMT CTN

for the CIBMTR • Scientific Director for the Autoimmune Diseases and Cellular

Therapies Working Committee as well as the Regimen-Related Toxicity and Supportive Care Working Committee

• Protocol Officer and Director of Medical Monitors for the BMT CTN • CIBMTR representative to the Worldwide Network for Blood and

Marrow Transplantation • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program

Roberta King, MPH • Vice President for CIBMTR Minneapolis • Oversees the administrative, scientific, and statistical support

activities of CIBMTR Minneapolis, which include research administration, human subject protection program, data management, auditing and monitoring, observational research, prospective research, and IT

• Staff Liaison to the NMDP/Be The Match Donor and Patient Safety Monitoring Advisory Group

Patricia Steinert, PhD, MBA • Administrator for CIBMTR Milwaukee • Oversees the administrative, scientific, and statistical support

activities of CIBMTR Milwaukee, which include data operations, development, observational research and prospective research, IT and meetings

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_Burns%20LJ.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_PasquiniM.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_KingR.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_SteinertP.pdf


Page | 81

SCIENTIFIC DIRECTORS

Mukta Arora, MD, MBBS, MS • Scientific Director of the Graft-versus-Host Disease Working

Committee • Associate Professor of Medicine at the University of Minnesota • Attending physician in the University of Minnesota HCT program

Anita D’Souza, MD • Assistant Scientific Director of the Plasma Cell Disorders and Adult

Solid Tumors Working Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program

Parameswaran Hari, MD, MS • Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors

Working Committee • Armand J. Quick – William F. Stapp Professor of Hematology at MCW • Section Head of Hematologic Malignancies and Adult BMT Program at

MCW • Attending physician in the MCW HCT program

Mehdi Hamadani, MD • Scientific Director of the Lymphoma Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_AroraM.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_D%27SouzaA.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_HariP.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_HamadaniM.pdf


Page | 82

Stephanie J. Lee, MD, MPH • Senior Scientific Director of Immunobiology Research for the CIBMTR • Scientific Director of the Immunobiology Working Committee • Professor of Medicine at the University of Washington • Attending physician in the Fred Hutchinson Cancer Research Center

HCT program

Martin Maiers, MS • Director of Bioinformatics Research for the CIBMTR • Staff liaison to the NMDP/Be The Match Histocompatibility Advisory

Group (also known as the CIBMTR Immunobiology Steering Committee)

• Co-Chair of Informatics: International Histocompatibility and Immunogenetics Workshop

• Member of World Health Organization HLA Nomenclature Committee

Marcie Riches, MD, MS • Scientific Director of the Infection and Immune Reconstitution

Working Committee • Protocol Officer and Medical Monitor for several BMT CTN trials • Clinical Associate Professor of Medicine at the University of North

Carolina at Chapel Hill • Director of BMT Clinical Research and Data Quality at the University of

North Carolina at Chapel Hill • Attending physician in the University of North Carolina Lineberger

Comprehensive Cancer Center HCT program

Wael Saber, MD, MS • Scientific Director of the Chronic Leukemia and Health Services and

International Studies Working Committees • Assistant Scientific Director of the Acute Leukemia Working

Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_LeeS.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_RichesM.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_SaberW.pdf


Page | 83

Stephen Spellman, MBS • Scientific Director of the Graft-versus-Host Disease and

Immunobiology Working Committees • Director of Immunobiology and Observational Research for the

CIBMTR • Principal Investigator for the Research Repository • Staff liaison to the NMDP/Be The Match Cord Blood Advisory Group

Research subcommittee and Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee)

• Program Manager for the NMDP/Be The Match Office of Naval Research Grant

Elizabeth Thiel, MD, MS • Scientific Director of the Pediatric Cancer Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW Palliative Care program

STATISTICAL DIRECTORS

Kwang Woo Ahn, PhD • Biostatistician for the Chronic Leukemia, Infection and Immune

Reconstitution, and Lymphoma Working Committees • Associate Professor of Biostatistics at MCW

Ruta Brazauskas, PhD • Biostatistician for the Health Services and International Studies

Working Committee as well as the Late Effects and Quality of Life and Autoimmune Disorders and Cellular Therapies Working Committees

• Assistant Professor of Biostatistics at MCW

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_SpellmanS.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_ThielE.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_WooAhn.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_BrazauskasR.pdf


Page | 84

Brent Logan, PhD • Biostatistician for the Donor Health and Safety Working Committee

and the Regimen-Related Toxicity and Supportive Care Working Committee

• Lead Statistician for the BMT CTN and Statistical Consultant to the NMDP/Be The Match

• Professor of Biostatistics at MCW • Director of the Division of Biostatistics at MCW

Tao Wang, PhD • Biostatistician for the Graft-versus-Host Disease and Immunobiology

Working Committees • Associate Professor of Biostatistics at MCW

OTHER LEADERSHIP STAFF

Erik Bergman, MBA, MS • Director of IT for the CIBMTR in Milwaukee • Leads the IT staff in Milwaukee, which is organized in four teams:

Database, Applications Development, Technology Services, and Project Management

• Oversees management of the Research Database, including extraction of data from source systems as well as their transformation and loading to the database

• Responsible for data retrievals from the Research Database as well as key solutions for sharing data with stakeholders

Janet Brunner-Grady, PA-C • Program Director of Data Operations for the CIBMTR • Manages the clinical research coordinators, develops training

programs, and monitors transplant center CPI • Assists clinical research coordinators on both campuses with clinical

transplant-related questions

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_LoganB.pdf

http://www.cibmtr.org/About/WhoWeAre/leaders/Documents/Bio_WangT.pdf


Page | 85

Debra Christianson • Senior Manager of Auditing and Monitoring for the CIBMTR • Oversees the monitoring program for RCI BMT clinical trials and the

on-site source document audits on data submitted to the Research Database

• Responsible for the FormsNet Instruction Manual

Rebecca Drexler • Senior Manager of Prospective Research for the CIBMTR • Manages the activities of the RCI BMT, including the Survey Research

Group • Oversees the administration of the Clinical Trials Advisory Committee

Sherry Fisher • Director of Advancement for the CIBMTR • Manages the advancement activities and the Corporate Program,

which generates new revenue to create a continued source of non-federal financial support

• Oversees meetings and communications activities, including the annual BMT Tandem Meetings

Marie Matlack • Senior Manager of Data Management for the CIBMTR • Manages clinical research coordinators and senior research

programmers as well as data entry and imaging staff • Oversees form revision and development and the CPI program • Collaborates with the Minneapolis IT staff to develop enhancements

to FormsNet


Page | 86

Waleska S. Pérez, MPH • Program Director of Statistics and Clinical Outcomes Research for the

CIBMTR • Oversees the Master’s-level statisticians of CIBMTR Milwaukee • Provides administrative oversight of the Clinical Outcomes Research

Program

Matt Prestegaard • Director of IT for the CIBMTR in Minneapolis • Leads the IT staff in Minneapolis, including project managers,

programmer analysts, business systems and data analysts, quality assurance analysts, metadata analysts, and managers

• Develops, implements, and supports CIBMTR electronic data management, capture, and messaging systems (FormsNet and AGNIS); data marts and data warehouses; infrastructure for these applications; and the curation of common data elements within the Cancer Data Standards Registry and Repository

CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP

Page | 87

APPENDIX C: COMMITTEE MEMBERSHIP

CIBMTR committees provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of its scientific and medical communities. All committees and their functions are listed in Table 1.3.

APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP

The Advisory Committee provides oversight for CIBMTR policies and scientific agenda and also partners with the Working Committees to prioritize scientific studies. Members are elected to three-year terms by the CIBMTR Assembly and must be from qualifying CRF transplant centers. All Advisory Committee terms begin on March 1.

ELECTED MEMBERS

Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC

VICE CHAIRS

North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX

Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom

Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil

Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia

MEMBERS AT LARGE

North America Steven Pavletic, MD, MS, NIH-NCI Experimental Transplantation and Immunology Branch, Bethesda, MD

Joseph Pidala, MD, MS, H Lee Moffitt Cancer Center and Research Institute, Tampa, FL

Michael Pulsipher, MD, Primary Children’s Hospital, Salt Lake City, UT

Bipin Savani, MD, Vanderbilt University Medical Center, Brentwood, TN

Marcel van den Brink, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY

Kirsten Williams, MD, Children’s National Medical Center, Washington, DC

Non-North America Ernst Holler, MD, Klinikum der Universitaet Regensburg, Germany

William Hwang, MBBS, MRCP, Singapore General Hospital, Singapore

Shinichiro Okamoto, MD, PhD, Keio University, Shinjuku-ku Tokyo, Japan


Page | 88

Miguel Sanz, Hospital Universitario La Fe, Valencia, Spain

Alok Srivastava, MD, Christian Medical College, Vellore, India

Afonso Vigorito, MD, PhD, Unicamp – Hemocentro, Campinas, Brazil

APPOINTED MEMBERS

ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada

Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA

Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE

Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI

Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX

Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN

Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee

Maureen Beaman, MBA, Chair, Consumer Advocacy Committee

EX OFFICIO MEMBERS

Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN

Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI

Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI

Senior Scientific Director for SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI

Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN

Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN

Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI

Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN

Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN

NMDP / MCW / HRSA Contracting Officer Representative

Shelley Grant, MHSA, Rockville, MD

NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)

MCW / HRSA Contracting Officer Representative

Christine Nishiguchi, MS, MPH, Rockville, MD


Page | 89

MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD

MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD

MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD

Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

Page | 90

APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

The Executive Committee, a subcommittee of the Advisory Committee, ensures that the organization carries out its mission and adheres to CIBMTR policies and procedures; it also provides advice and counsel to the Coordinating Center between meetings of the Advisory Committee. Specifically, the Executive Committee is responsible for providing scientific and policy advice to the Chief Scientific Director and Coordinating Center, reviewing audit results and making recommendations for improvement, and appointing a CIBMTR Co-Chair and additional CIBMTR representatives to the BMT Tandem Meetings Scientific Organizing Committee for the annual BMT Tandem Meetings. All Executive Committee terms begin on March 1.

ELECTED MEMBERS

Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC

VICE CHAIRS

North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX

Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom

Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil

Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia

APPOINTED MEMBERS

ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada

Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA

Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE

Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI

Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX

Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN

Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee

Maureen Beaman, MBA, Chair, Consumer Advocacy Committee

CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

Page | 91

EX OFFICIO MEMBERS

Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN

Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI

Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI

Senior Scientific Director for SCTOD

J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI

Associate Scientific Director CIBMTR Minneapolis

Dennis Confer, MD, CIBMTR, Minneapolis, MN

Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN

Research Administrator CIBMTR Milwaukee

Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI

Vice President CIBMTR Minneapolis

Roberta King, CIBMTR, Minneapolis, MN

Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN

NMDP / MCW /HRSA Contracting Officer Representative

Shelley Grant, MHSA, Rockville, MD

NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)

MCW / HRSA Contracting Officer Representative

Christine Nishiguchi, MS, MPH, Rockville, MD

MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD

MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD

MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD

Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

CIBMTR 2015 Annual Report APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP

Page | 92

APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP

The Consumer Advocacy Committee communicates research results and data to the non-medical community and provides patient and donor perspectives during the development of the CIBMTR research agenda. Many committee members have personal experience as a donor, recipient, or family member of a recipient.

CHAIRS James Omel, MD

Maureen Beaman, MBA

MEMBERS

Beatrice Abetti, MSW, Leukemia and Lymphoma Society

Jack Aiello, MS

Gerardo Camarillo, JD, MD Anderson Cancer Center

Jeffrey Haertling, ARK Air Express

Hilary Hall, Dana-Farber Cancer Institute

Barry Schatz, Cardinal Bernardin Cancer Center, Loyola University Medical Center

Kristin Scheeler, University of Wisconsin Hospital and Clinics

SCIENTIFIC DIRECTOR

J. Douglas Rizzo, MD, MS, CIBMTR

EX OFFICIO MEMBERS

Robyn Ashton, RN, MSN, HRSA

Jeffrey Chell, MD, NMDP/Be The Match

Dennis Confer, MD, NMDP/Be The Match

Ellen Denzen, (NMDP/Be The Match liaison) NMDP/Be The Match

Carol Doleysh, (CIBMTR liaison) CIBMTR

Rebecca Drexler, CIBMTR

Jessica Gillis-Smith, MPH, CIBMTR

Shelley Grant, MHSA, HRSA

Darlene Haven, NMDP/Be The Match

Mary Horowitz, MD, MS, CIBMTR

Elizabeth Murphy, EdD, RN, (NMDP/Be The Match liaison) NMDP/Be The Match

Christine Nishiguchi, MS, MPH, HRSA

Patricia Steinert, PhD, MBA, CIBMTR

CIBMTR 2015 Annual Report APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP

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APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP

The Nominating Committee consists of five members elected by the CIBMTR Assembly. Following an annual call for nominations, the Nominating Committee prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees. Elections are held annually by confidential electronic ballot. The Nominating Committee also makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments after seeking recommendations from the CIBMTR Assembly, Advisory Committee, and incumbent Working Committee Chairs. All terms begin on March 1.

CHAIR

Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

MEMBERS

Daniel Couriel, MD, University of Michigan, Ann Arbor, MI

Richard Champlin, MD, MD Anderson Cancer Center, Houston TX

Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

David Vesole, MD, PhD, Hackensack University Medical Center, Hackensack, NJ

CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP

Page | 94

APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP

For information on Scientific Working Committee structure and organization, see Section 1.3.1. For information on Working Committee studies and their accomplishments, see Section 2.1.1.

ACUTE LEUKEMIA WORKING COMMITTEE

Chairs Marcos de Lima, MD, University Hospitals Case Medical Center Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Hanna Khoury, MD, Emory University Hospital

Scientific Director Daniel Weisdorf, MD, CIBMTR Asst Scientific Dir Wael Saber, MD, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Hailin Wang, MPH, CIBMTR

AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE

Chairs Mitchell Cairo, MD, New York Medical College Ian Lewis, MBBS, PhD, Royal Adelaide Hospital David McKenna, MD, University of Minnesota Medical Center, Fairview

Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Kyle Bo-Subait, MPH, CIBMTR

CHRONIC LEUKEMIA WORKING COMMITTEE

Chairs Edwin Alyea, MD, Dana Farber Cancer Institute Uday Popat, MD, MD Anderson Cancer Center Ronald Sobecks, MD, Cleveland Clinic Foundation

Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Zhenhuan Hu, MS, CIBMTR

DONOR HEALTH AND SAFETY WORKING COMMITTEE

Chairs Paul O’Donnell, MD, PhD, Fred Hutchinson Cancer Research Center Michael Pulsipher, MD, Primary Children’s Hospital Galen Switzer, PhD, University of Pittsburgh Medical Center - Cancer Center Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Ex Officio Sr Advisor Dennis Confer, MD, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statisticians Deidre Kiefer, MPH, CIBMTR Pintip Chitphakdithai, PhD, CIBMTR Consumer Advocacy Committee Representatives

Beatrice Abetti, MSW, Leukemia and Lymphoma Society Maureen Beaman, MBA Jeffrey Haertling, ARK Air Express


Page | 95

GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE

Chairs Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center Vanderson Rocha, MD, PhD, Churchill Hospital Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside

Hospital Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Andrew St. Martin, MS, CIBMTR

GRAFT-VERSUS-HOST DISEASE WORKING COMMITTEE

Chairs Daniel Couriel, MD, University of Michigan Amin Alousi, MD, MD Anderson Cancer Center Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute

Scientific Directors Mukta Arora, MD, MS, CIBMTR Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Hemmer, MS, CIBMTR Consumer Advocacy Committee Representatives

Hilary Hall, Dana-Farber Cancer Institute James Omel, MD

HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE

Chairs Yoshiko Atsuta, MD, PhD, Nagoya University Graduate School of Medicine Carmem Bonfim, MD, Hospital de Clinicas Curitiba Jignesh Dalal, MD, The Children’s Mercy Hospitals and Clinics Theresa Hahn, PhD, Roswell Park Cancer Institute Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital

Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Naya He, MPH, CIBMTR Consumer Advocacy Committee Representatives

Jack Aiello, MS Gerardo Camarillo, JD, MD Anderson Cancer Center

IMMUNOBIOLOGY WORKING COMMITTEE

Chairs Michael Verneris, MD, University of Minnesota Medical Center, Fairview Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center

Scientific Directors Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Haagenson, MS, CIBMTR


Page | 96

INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE

Chairs Jeffery Auletta, MD, Nationwide Children’s Hospital Caroline Lindemans, MD, PhD, University Medical Center Utrecht Krishna Komanduri, MD, University of Miami

Scientific Director Marcie Riches, MD, MS, CIBMTR, H Lee Moffitt Cancer Center and Research Institute

Statistical Directors Kwang Woo Ahn, PhD, CIBMTR Soyoung Kim, PhD, CIBMTR

Statistician Min Chen, MS, CIBMTR

LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE

Chairs Bipin Savani, MD, Vanderbilt University Medical Center Mary Flowers, MD, Fred Hutchinson Cancer Research Center Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute - NIH

Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR Consumer Advocacy Committee Representative

Barry Schatz, Loyola University Medical Center

LYMPHOMA WORKING COMMITTEE

Chairs Sonali Smith, MD, University of Chicago Hospitals Anna Sureda, MD, PhD Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital

Scientific Director Mehdi Hamadani, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Alyssa DiGilio, MS, CIBMTR

PEDIATRIC CANCER WORKING COMMITTEE

Chairs Gregory Hale, MD, All Children’s Hospital Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center Angela Smith, MD, MS, University of Minnesota Medical Center, Fairview

Scientific Director Elizabeth Thiel, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR


Page | 97

PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE

Chairs Amrita Krishnan, MD, City of Hope National Medical Center Cristina Gasparetto, MD, Duke University Medical Center Yago Nieto, MD, PhD, MD Anderson Cancer Center Tomer Mark, MD, New York Presbyterian Hospital at Cornell

Scientific Director Parameswaran Hari, MD, MS, CIBMTR Asst Scientific Dir Anita D’Souza, MD, CIBMTR Statistical Directors Mei-Jie Zhang, PhD, CIBMTR Raphael Fraser, PhD, CIBMTR Statistician Jiaxing Huang, MS, CIBMTR Consumer Advocacy Committee Representative James Omel, MD

PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND OTHER NON-MALIGNANT MARROW DISORDERS WORKING COMMITTEE

Chairs Paolo Anderlini, MD, MD Anderson Cancer Center Neena Kapoor, MD, Children’s Hospital of Los Angeles Jaap-Jan Boelens, MD, PhD, University Medical Center Utrecht Vikram Mathews, MD, Christian Medical College Hospital

Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician TBD

REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE

Chairs Andrew Artz, MD, MS, University of Chicago Hospitals Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania

Medical Center Shin Mineishi, MD, University of Alabama at Birmingham

Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statistician Xiaochun Zhu, MS, CIBMTR

CIBMTR 2015 Annual Report APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP

Page | 98

APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP

The NMDP/Be The Match Histocompatibility Advisory Group also serves as the CIBMTR Immunobiology Steering Committee. This committee reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies.

CHAIR

Carolyn K. Hurley, PhD, Diplomate ABHI, Georgetown University Hospital

ADVISORY GROUP MEMBERS

Juliet Barker, MD, Memorial Sloan Kettering Cancer Center

Sarah Cooley, MD, University of Minnesota

Mary Eapen, MD, MS, Medical College of Wisconsin

Marcelo Fernandez-Viña, PhD, Stanford Hospital and Clinics

Brent Logan, PhD, Medical College of Wisconsin

Carlheinz Mueller, MD, PhD, German National Bone Marrow Donor Registry (ZKRD)

Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center

David Porter, MD, Abramson Cancer Center University of Pennsylvania Medical Center

Raja Rajalingham, PhD, University of California, San Francisco

Bronwen Shaw, MD, PhD, Medical College of Wisconsin

EX OFFICIO MEMBERS

Dennis Confer, MD, NMDP/Be The Match

Jason Dehn, MPH, NMDP/Be The Match

Karen Dodson, NMDP/Be The Match

Melissa Greenwald, MD, HRSA

Robert Hartzman, MD, Capt. MC, USN (Ret.), Navy Representative, C.W. Bill Young Marrow Donor Recruitment and Research Program

Martin Maiers, CIBMTR

Stephen Spellman, MBS, CIBMTR

John Wingard, MD, NMDP/Be The Match Board Chair

CIBMTR 2015 Annual Report APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP

Page | 99

APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP

The Clinical Trials Advisory Committee assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT (Section 2.3.2).

CHAIR

John Levine, MD, MS, Mount Sinai Medical Center

MEMBERS

Colleen Delaney, MD, MSc, Fred Hutchinson Cancer Research Center

Marcos de Lima, MD, University Hospitals Case Medical Center

Hugo Fernandez, MD, H Lee Moffitt Cancer Center and Research Institute

John Koreth, MBBS, DPhil, Dana Farber Cancer Institute

Mark Litzow, MD, Mayo Clinic Rochester

Margaret MacMillan, MD, MSc, University of Minnesota Medical Center, Fairview

Sophie Paczesny, MD, PhD, Indiana University Hospital/Riley Hospital for Children

Katayoun Rezvani, MD, PhD, M.D. Anderson Cancer Center

Jan Storek, MD, PhD, University of Calgary

APPOINTED MEMBERS

Maureen Beaman, MBA

James Omel, MD

EX OFFICIO MEMBERS

Dennis Confer, MD, CIBMTR

Rebecca Drexler, BS, CIBMTR

Robert Hartzman, MD, Capt MC, USN (Ret.)

Mary Horowitz, MD, MS, CIBMTR

Roberta King, CIBMTR

Brent Logan, PhD, CIBMTR

Nancy Poland, MA, NMDP/Be The Match Operations

Marcie Riches, MD, MS, The University of North Carolina at Chapel Hill

Kevin Weber, NMDP/Be The Match Operations

Daniel Weisdorf, MD, CIBMTR

CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

Page | 100

APPENDIX D: PUBLICATIONS

The PMCID number is assigned by PubMed Central, the NIH’s free digital archive of biomedical and life sciences journal literature, and is in compliance with the NIH policy on public access.

APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

The following publications were generated by Scientific Working Committees within the Clinical Outcomes Research Program. For more information about the Working Committees, see Section 2.1.1.

SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

Authors Title Citation PMCID

Logan AC, Wang Z, Alimoghaddam K, Wong RM, Lai T, Negrin RS, Grumet C, Logan BR, Zhang M-J, Spellman SR, Lee SJ, Miklos DB

ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):746-754. doi:10.1016/j.bbmt. 2014.12.036. Epub 2015 Jan 5

PMC4363312

Gadalla SM, Wang T, Haagenson M, Spellman SR, Lee SJ, Williams KM, Wong JY, De Vivo I, Savage SA

Association between donor leukocyte telomere length and survival after unrelated allogeneic hematopoietic cell transplantation for severe aplastic anemia

JAMA: The Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. doi:10.1001/ jama.2015.7. Epub 2015 Feb 10

PMC4388056

Chen Y-B, Lane AA, Logan BR, Zhu X, Akpek G, Aljurf MD, Artz AS, Bredeson CN, Cooke KR, Ho VT, Lazarus HM, Olsson RF, Saber W, McCarthy PL, Pasquini MC

Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous hematopoietic cell transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. doi:10.1016/ j.bbmt.2015.02.005. Epub 2015 Feb 13

PMC4426014


Page | 101

SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID

Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M

Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning

British Journal of Haematology. 2015 Jun 1; 169(5):711-718. doi:10.1111/bjh.13347. Epub 2015 Mar 27

PMC4433436

Inamoto Y, Shah NN, Savani BN, Shaw BE, Abraham AA, Ahmed IA, Akpek G, Atsuta Y, Baker KS, Basak GW, Bitan M, DeFilipp Z, Gregory TK, Greinix HT, Hamadani M, Hamilton BK, Hayashi RJ, Jacobsohn DA, Kamble RT, Kasow KA, Khera N, Lazarus HM, Malone AK, Lupo-Stanghellini MT, Margossian SP, Muffly LS, Norkin M, Ramanathan M, Salooja N, Schoemans H, Wingard JR, Wirk B, Wood WA, Yong A, Duncan CN, Flowers MED, Majhail NS

Secondary solid cancer screening following hematopoietic cell transplantation

Bone Marrow Transplantation. 2015 Aug 1; 50(8):1013-1023. doi:10.1038/ bmt.2015.63. Epub 2015 Mar 30

N/A

Holter-Chakrabarty JL, Pierson N, Zhang M-J, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL

The sequence of cyclophosphamide and myeloablative total body irradiation in hematopoietic cell transplantation for patients with acute leukemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1251-1257. doi:10.1016/j.bbmt. 2015.03.017. Epub 2015 Mar 31

PMC4465990


Page | 102


Sorror ML, Logan BR, Zhu X, Rizzo JD, Cooke KR, McCarthy PL, Ho VT, Horowitz MM, Pasquini MC

Prospective validation of the predictive power of the hematopoietic cell transplantation comorbidity index: a Center for International Blood and Marrow Transplant Research study

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479-1487. doi:10.1016/ j.bbmt.2015.04.004. Epub 2015 Apr 7

PMC4512746

Mehta PA, Zhang M-J, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel-Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM

Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1273-1277. doi:10.1016/j.bbmt. 2015.04.008. Epub 2015 Apr 10

PMC4465998

Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OTH, Pasquini MC, Schriber JR, Cooke KR

Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies

Leukemia. 2015 Aug 1; 29(8):1754-1762. doi:10.1038/leu. 2015.75. Epub 2015 Apr 24

PMC4527886

Goyal SD, Zhang MJ, Wang HL, Akpek G, Copelan EA, Freytes C, Gale RP, Hamadani M, Inamoto Y, Kamble RT, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Reshef R, Ritchie DS, Saber W, Savani BN, Seber A, Shea TC, Tallman MS, Wirk B, Bunjes DW, Devine SM, de Lima M, Weisdorf DJ, Uy GL

Allogeneic hematopoietic cell transplant for AML: no impact of pre-transplant extramedullary disease on outcome

Bone Marrow Transplantation. 2015 Aug 1; 50(8):1057-1062. doi:10.1038/ bmt.2015.82. Epub 2015 Apr 27

PMC4527880


Page | 103


Sobecks RM, Wang T, Askar M, Gallagher MM, Haagenson M, Spellman S, Fernandez-Vina M, Malmberg K-J, Müller C, Battiwalla M, Gajewski J, Verneris MR, Ringdén O, Marino S, Davies S, Dehn J, Bornhäuser M, Inamoto Y, Woolfrey A, Shaw P, Pollack M, Weisdorf D, Milller J, Hurley C, Lee SJ, Hsu K

Impact of KIR and HLA genotypes on outcomes after reduced-intensity conditioning hematopoietic cell transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1589-1596. doi:10.1016/ j.bbmt.2015.05.002. Epub 2015 May 8

PMC4537837

Bachanova V, Burns LJ, Ahn KW, Laport GG, Akpek G, Kharfan-Dabaja MA, Nishihori T, Agura E, Armand P, Jaglowski SM, Cairo MS, Cashen AF, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Ghosh N, Holmberg LA, Inwards DJ, Kanate AS, Lazarus HM, Malone AK, Munker R, Mussetti A, Norkin M, Prestidge TD, Rowe JM, Satwani P, Siddiqi T, Stiff PJ, William BM, Wirk B, Maloney DG, Smith SM, Sureda AM, Carreras J, Hamadani M

Impact of pretransplantation 18F-fluorodeoxy glucose-positron emission tomography status on outcomes after allogeneic hematopoietic cell transplantation for non-Hodgkin Lymphoma


PMC4558181


Page | 104


Urbano-Ispizua A, Pavletic SZ, Flowers ME, Klein JP, Zhang M-J, Carreras J, Montoto S, Perales M-A, Aljurf MD, Akpek G, Bredeson CN, Costa LJ, Dandoy C, Freytes CO, Fung HC, Gale RP, Gibson J, Hamadani M, Hayashi RJ, Inamoto Y, Inwards DJ, Lazarus HM, Maloney DG, Martino R, Munker R, Nishihori T, Olsson RF, Rizzieri DA, Reshef R, Saad A, Savani BN, Schouten HC, Smith SM, Socié G, Wirk B, Yu LC, Saber W

The impact of graft-versus-host disease on the relapse rate in patients with lymphoma depends on the histological subtype and the intensity of the conditioning regimen

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1746-1753. doi:10.1016/ j.bbmt.2015.05.010. Epub 2015 May 15

PMC4568162

Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, Al-Seraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Diaz Perez MA, Veys PA, Eapen M

Hematopoietic stem cell transplantation for infantile osteopetrosis

Blood. 2015 Jul 9; 126(2):270-276. doi:10.1182/blood-2015-01-625541. Epub 2015 May 26

PMC4497967

Hahn T, Sucheston-Campbell LE, Preus L, Zhu X, Hansen JA, Martin PJ, Yan L, Liu S, Spellman S, Tritchler D, Clay A, Onel K, Pasquini M, McCarthy PL

Establishment of definitions and review process for consistent adjudication of cause-specific mortality after allogeneic unrelated-donor hematopoietic cell transplantation


PMC4537799


Page | 105


Inamoto Y, Flowers MED, Wang T, Urbano-Ispizua A, Hemmer MT, Cutler CS, Couriel DR, Alousi AM, Antin JH, Gale RP, Gupta V, Hamilton BK, Kharfan-Dabaja MA, Marks DI, Ringdén OTH, Socié G, Solh MM, Akpek G, Cairo MS, Chao NJ, Hayashi RJ, Nishihori T, Reshef R, Saad A, Shah A, Teshima T, Tallman MS, Wirk B, Spellman SR, Arora M, Martin PJ

Tacrolimus versus cyclosporine after hematopoietic cell transplantation for acquired aplastic anemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1776-1782. doi:10.1016/ j.bbmt.2015.05.023. Epub 2015 May 30

PMC4568149

Verneris MR, Lee SJ, Ahn KW, Wang H-L, Battiwalla M, Inamoto Y, Fernandez-Vina MA, Gajewski J, Pidala J, Munker R, Aljurf M, Saber W, Spellman S, Koreth J

HLA mismatch Is associated with worse outcomes after unrelated donor reduced-intensity conditioning hematopoietic cell transplantation: an analysis from the Center for International Blood and Marrow Transplant Research

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783-1789. doi:10.1016/ j.bbmt.2015.05.028. Epub 2015 Jun 6

PMC4568127

Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ

Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial


PMC4568172


Page | 106


Ayas M, Eapen M, Le-Rademacher J, Carreras J, Abdel-Azim H, Alter BP, Anderlini P, Battiwalla M, Bierings M, Buchbinder DK, Bonfim C, Camitta BM, Fasth AL, Gale RP, Lee MA, Lund TC, Myers KC, Olsson RF, Page KM, Prestidge TD, Radhi M, Shah AJ, Schultz KR, Wirk B, Wagner JE, Deeg HJ

Second allogeneic hematopoietic cell transplantation for patients with Fanconi anemia and bone marrow failure


PMC4568139

Shaw BE, Logan BR, Kiefer DM, Chitphakdithai P, Pedersen TL, Abdel-Azim H, Abidi MH, Akpek G, Diaz MA, Artz AS, Dandoy C, Gajewski JL, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Majhail NS, O'Donnell PV, Olsson RF, Savani BN, Schears RM, Stroncek DF, Switzer GE, Williams EP, Wingard JR, Wirk BM, Confer DL, Pulsipher MA

Analysis of the effect of race, socioeconomic status, and center size on unrelated National Marrow Donor Program donor outcomes: donor toxicities are more common at low-volume bone marrow collection centers


PMC4568129

Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales M-A, Pingali SR, Porter DL, Riches MR, Ringdén OTH, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M

Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia

Blood. 2015 Aug 20; 126(8):1033-1040. doi:10.1182/blood-2015-04-639831. Epub 2015 Jun 30

PMC4543223


Page | 107


Satwani P, Ahn KW, Carreras J, Abdel-Azim H, Cairo MS, Cashen A, Chen AI, Cohen JB, Costa LJ, Dandoy C, Fenske TS, Freytes CO, Ganguly S, Gale RP, Ghosh N, Hertzberg MS, Hayashi RJ, Kamble RT, Kanate AS, Keating A, Kharfan-Dabaja MA, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Prestidge TD, Rolon JM, Savani BN, Vose JM, Wood WA, Inwards DJ, Bachanova V, Smith SM, Maloney DG, Sureda A, Hamadani M

A prognostic model predicting autologous transplantation outcomes in children, adolescents and young adults with Hodgkin lymphoma

Bone Marrow Transplantation. 2015 Nov 1; 50(11):1416-1423. doi:10.1038/ bmt.2015.177. Epub 2015 Aug 3

PMC4633349

Klyuchnikov E, Bacher U, Kröger NM, Hari PN, Ahn KW, Carreras J, Bachanova V, Bashey A, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Hertzberg MS, Holmberg LA, Kharfan-Dabaja MA, Klein A, Ku GH, Laport GG, Lazarus HM, Miller AM, Mussetti A, Olsson RF, Slavin S, Usmani SZ, Vij R, Wood WA, Maloney DG, Sureda AM, Smith SM, Hamadani M

Reduced-intensity allografting as first transplantation approach in relapsed/refractory grades one and two follicular lymphoma provides improved outcomes in long-term survivors

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2091-2099. doi:10.1016/ j.bbmt.2015.07.028. Epub 2015 Aug 4

PMC4639453

Petersdorf EW, Malkki M, O'hUigin C, Carrington M, Gooley T, Haagenson MD, Horowitz MM, Spellman SR, Wang T, Stevenson P

High HLA-DP expression and graft-versus-host disease

New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. doi:10.1056/ NEJMoa1500140. Epub 2015 Aug 13

PMC4560117

Knight JM, Rizzo JD, Logan BR, Wang T, Arevalo JM, Ma J, Cole SW

Low socioeconomic status, adverse gene expression profiles, and clinical outcomes in hematopoietic stem cell transplant recipients

Clinical Cancer Research. Epub 2015 Aug 18

[PMC Journal – In Process]


Page | 108


Uy GL, Costa LJ, Hari PN, Zhang M-J, Huang J-X, Anderson KC, Bredeson CN, Callander NS, Cornell RF, Perez MAD, Dispenzieri A, Freytes CO, Gale RP, Garfall A, Gertz MA, Gibson J, Hamadani M, Lazarus HM, Kalaycio ME, Kamble RT, Kharfan-Dabaja MA, Krishnan AY, Kumar SK, Kyle RA, Landau HJ, Lee CH, Maiolino A, Marks DI, Mark TM, Munker R, Nishihori T, Olsson RF, Ramanathan M, Rodriguez TE, Saad AA, Savani BN, Schiller GJ, Schouten HC, Schriber JR, Scott E, Seo S, Sharma M, Ganguly S, Stadtmauer EA, Tay J, To LB, Vesole DH, Vogl DT, Wagner JL, Wirk B, Wood WA, D'Souza A

Contribution of chemotherapy mobilization to disease control in multiple myeloma treated with autologous hematopoietic cell transplantation

Bone Marrow Transplantation. doi:10.1038/ bmt.2015.190. Epub 2015 Aug 24

PMC4548821

Burke MJ, Verneris MR, Le Rademacher J, He W, Abdel-Azim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL

Transplant outcomes for children with T cell acute lymphoblastic leukemia in second remission: a report from the Center for International Blood and Marrow Transplant Research


PMC4654112


Page | 109


Ponce DM, Eapen M, Sparapani R, O'Brien TA, Chan KW, Chen J, Craddock J, Schultz KR, Wagner JE, Perales M-A, Barker JN

In vivo T cell depletion with myeloablative regimens on outcomes after cord blood transplantation for acute lymphoblastic leukemia in children


PMC4639413

Pasquini MC, Zhang M-J, Medeiros BC, Armand P, Hu Z-H, Nishihori T, Aljurf MD, Akpek G, Cahn J-Y, Cairo MS, Cerny J, Copelan EA, Deol A, Freytes CO, Gale RP, Ganguly S, George B, Gupta V, Hale GA, Kamble RT, Klumpp TR, Lazarus HM, Luger SM, Liesveld JL, Litzow MR, Marks DI, Martino R, Norkin M, Olsson RF, Oran B, Pawarode A, Pulsipher MA, Ramanathan M, Reshef R, Saad AA, Saber W, Savani BN, Schouten HC, Ringdén O, Tallman MS, Uy GL, Wood WA Jr, Wirk B, Pérez WS, Batiwalla M, Weisdorf DJ

Hematopoietic cell transplantation outcomes in monosomal karyotype myeloid malignancies

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.08.024. Epub 2015 Aug 29


Jindra PT, Conway SE, Ricklefs SM, Porcella SF, Anzick SL, Haagenson M, Wang T, Spellman S, Milford E, Kraft P, McDermott DH, Abdi R

Analysis of a genetic polymorphism in the costimulatory molecule TNFSF4 with HSCT outcomes

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.08.037. Epub 2015 Sep 5



Page | 110


D'Souza A, Dispenzieri A, Wirk B, Zhang M-J, Huang J, Gertz MA, Kyle RA, Kumar S, Comenzo RL, Gale RP, Lazarus HM, Savani BN, Cornell RF, Weiss BM, Vogl DT, Freytes CO, Scott EC, Landau HJ, Moreb JS, Costa LJ, Ramanathan M, Callander NS, Kamble RT, Olsson RF, Ganguly S, Nishihori T, Kindwall-Keller TL, Wood WA, Mark TM, Hari P

Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a Center for International Blood and Marrow Transplant Research study

Journal of Clinical Oncology. 2015 Nov 10; 33(32):3741-3749. doi:10.1200/JCO. 2015.62.4015. Epub 2015 Sep 14


Ehrhardt MJ, Brazauskas R, He W, Rizzo JD, Shaw BE

Survival of patients who develop solid tumors following hematopoietic stem cell transplantation

Bone Marrow Transplantation. doi:10.1038/bmt. 2015.203. Epub 2015 Sep 14

PMC4570237

Klyuchnikov E, Bacher U, Woo Ahn K, Carreras J, Kröger NM, Hari PN, Ku GH, Ayala E, Chen AI, Chen Y-B, Cohen JB, Freytes CO, Gale RP, Kamble RT, Kharfan-Dabaja MA, Lazarus HM, Martino R, Mussetti A, Savani BN, Schouten HC, Usmani SZ, Wiernik PH, Wirk B, Smith SM, Sureda A, Hamadani M

Long-term survival outcomes of reduced-intensity allogeneic or autologous transplantation in relapsed grade 3 follicular lymphoma

Bone Marrow Transplantation. doi:10.1038/bmt. 2015.223. Epub 2015 Oct 5


Kollman C, Spellman SR, Zhang M-J, Hassebroek A, Anasetti C, Antin JH, Champlin RE, Confer D, DiPersio JF, Fernandez-Viña M, Hartzman RJ, Horowtiz MM, Hurley CK, Karanes C, Maiers M, Mueller CR, Perales M-A, Setterholm M, Woolfrey AE, Yu N, Eapen M

The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy

Blood. doi:10.1182/blood-2015-08-663823. Epub 2015 Nov 2



Page | 111


Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papinicolou GA, Wingard JR, Young J-AH, Bennett CL

Risk factors and impact of non-Aspergillus mold infections following allogeneic HCT: a CIBMTR infection and immune reconstitution analysis

Bone Marrow Transplantation. doi:10.1038/bmt. 2015.263. Epub 2015 Nov 2


Anthias C, Shaw BE, Kiefer DM, Liesveld JL, Yared J, Kamble RT, D'Souza A, Hematti P, Seftel MD, Norkin M, DeFilipp ZM, Kasow KA, Abidi MH, Savani BN, Shah NN, Anderlini P, Diaz MA, Malone AK, Halter JP, Lazarus HM, Logan BR, Switzer GE, Pulsipher MA, Confer DL, O'Donnell PV

Significant improvements in the practice patterns of adult related donor care in US transplant centers

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.11.008. Epub 2015 Nov 18


Kanate AS, Mussetti A, Kharfan-Dabaja MA, Ahn KW, DiGilio A, Beitinjaneh A, Chhabra S, Fenske TS, Freytes C, Gale RP, Ganguly S, Hertzberg M, Klyuchnikov E, Lazarus HM, Olsson R, Perales M-A, Rezvani A, Riches M, Saad A, Slavin S, Smith SM, Sureda A, Yared J, Ciurea S, Armand P, Salit R, Bolaños-Meade J, Hamadani M

Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched unrelated donors

Blood. doi:10.1182/ blood-2015-09-671834. Epub 2015 Dec 15


Seftel MD, Neuberg D, Zhang M-J, Wang HL, Ballen KK, Bergeron J, Couban S, Freytes CO, Hamadani M, Kharfan-Dabaja MA, Lazarus HM, Nishihori T, Paulson K, Saber W, Sallan SE, Soiffer R, Tallman MS, Woolfrey AE, DeAngelo DJ, Weisdorf DJ

Pediatric-inspired therapy compared to allografting for Philadelphia chromosome negative adult ALL in first complete remission

American Journal of Hematology. doi:10.1002/ajh.24285. Epub 2015 Dec 23


CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS

Page | 112

APPENDIX D2: BMT CTN PUBLICATIONS

The following publications were generated by the BMT CTN, a component of the Clinical Trials Support Program, which conducts multi-institutional Phase II and III trials focused on HCT. The BMT CTN Data and Coordinating Center maintains continuity of operations and facilitates effective communications. The Data and Coordinating Center effort is a collaboration of the CIBMTR, NMDP/Be The Match, and the Emmes Corporation. For more information, see Section 2.3.1.

BMT CTN PUBLICATIONS Authors Title Citation PMCID

(2014 publication not previously reported) Levine JE, Braun TM, Harris AC, Holler E, Taylor A, Miller H, Magenau J, Weisdorf DJ, Ho VT, Bolaños-Meade J, Alousi AM, Ferrara JLM

A prognostic score for acute graft-versus-host disease based on biomarkers: a multicentre study

The Lancet Haematology. 2(1):e21-e29. doi:10.1016/S2352-3026(14)00035-0. Epub 2014 Dec 23

PMC4340092

MacMillan ML, Robin M, Harris AC, DeFor TE, Martin PJ, Alousi A, Ho VT, Bolaños-Meade J, Ferrara JLM, Jones R, Arora M, Blazar BR, Holtan SG, Jacobsohn D, Pasquini M, Socie G, Antin JH, Levine JE, Weisdorf DJ

A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. doi:10.1016/j.bbmt. 2015.01.001. Epub 2015 Jan 10

PMC4359643

Holtan SG, Verneris MR, Schultz KR, Newell LF, Meyers G, He F, DeFor TE, Vercellotti GM, Slungaard A, MacMillan ML, Cooley SA, Blazar BR, Panoskaltsis-Mortari A, Weisdorf DJ

Circulating angiogenic factors associated with response and survival in patients with acute graft-versus-host disease: results from Blood and Marrow Transplant Clinical Trials Network 0302 and 0802

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1029-1036. doi:10.1016/ j.bbmt.2015.02.018. Epub 2015 Mar 7

PMC4426052


Page | 113


Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ

Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial


PMC4568172

Anderlini P, Wu J, Gersten I, Ewell M, Tolar J, Antin JH, Adams R, Arai S, Eames G, Horwitz ME, McCarty J, Nakamura R, Pulsipher MA, Rowley S, Leifer E, Carter SL, DiFronzo NL, Horowitz MM, Confer D, Deeg HJ, Eapen M

Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study

Lancet Haematology. 2015 Sep 1; 2(9):e367-375. doi:10.1016/ S2352-3026(15)00147-7. Epub 2015 Sep 2


Young JAH, Logan BR, Wu J, Wingard JR, Weisdorf DJ, Mudrick C, Knust K, Horowitz MM, Confer DL, Dubberke ER, Pergam SA, Marty FM, Strasfeld LM, Brown JM, Langston AA, Schuster MG, Kaul DR, Martin SI, Anasetti C

Infections following transplantation of bone marrow or peripheral-blood stem cells from unrelated donors

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.09.013. Epub 2015 Sep 23


Wood WA, Le-Rademacher J, Syrjala KL, Jim H, Jacobsen PB, Knight JM, Abidi MH, Wingard JR, Majhail NS, Geller NL, Rizzo JD, Fei M, Wu J, Horowitz MM, Lee SJ

Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902)

Cancer. doi: 10.1002/ cncr.29717. Epub 2015 Oct 6



Page | 114


Giralt S, Garderet L, Durie B, Cook G, Gahrton G, Bruno B, Hari P, Lokhorst H, McCarthy P, Krishnan A, Sonneveld P, Goldschmidt H, Jagannath S, Barlogie B, Mateos M, Gimsing P, Sezer O, Mikhael J, Jin L, Dimopoulos M, Mazumder A, Palumbo A, Abonour R, Anderson K, Attal M, Blade J, Bird J, Cavo M, Comenzo R, de la Rubia J, Einsele H, Sanz RG, Hillengass J, Holstein S, Johnsen H, Joshua D, Koehne G, Kumar S, Kyle R, Leleu X, Lonial S, Ludwig H, Nahi H, Nooka A, Orlowski R, Rajkumar V, Reiman A, Richardson P, Rivas E, San Miguel J, Turreson I, Usmani S, Vesole D, Bensinger W, Qazilbash M, Efebera Y, Mohty M, Gasparreto C, Gajewski J, LeMaistre C, Bredeson C, Moreau P, Pasquini M, Kroeger N, Stadtmauer E

American Society of Blood and Marrow Transplant, European Society of Blood and Marrow Transplantation, Blood and Marrow Transplant Clinical Trials Network and International Myeloma Working Group Consensus Conference on Salvage Hematopoietic Cell Transplantation in Patients with Relapsed Multiple Myeloma

Biology of Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2039-2051. doi: 10.1016/ j.bbmt.2015.09.016. Epub 2015 Sep 30


Devine SM, Owzar K, Blum W, Mulkey F, Stone RM, Hsu JW, Champlin RE, Chen Y-B, Vij R, Slack J, Soiffer RJ, Larson RA, Shea TC, Hars V, Sibley AB, Geralt S, Carter S, Horowitz MM, Linker C, Alyea EP

Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: results from cancer and leukemia group B 100103 (alliance for clinical trials in oncology)/blood and marrow transplant clinical trial network 0502

Journal of Clinical Oncology. Epub 2015 Nov 2


CIBMTR 2015 Annual Report APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS

Page | 115

APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS

The following publications were generated by the Health Services Research program, through which the CIBMTR conducts research in health disparities, health policy, and system capacity issues involving HCT. For more information, see Section 2.4.

HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS


Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP

Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems

Journal of Oncology Practice. 11(2):e120-e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1

PMC4371120

Majhail NS, Mau L-W, Chitphakdithai P, Payton T, Eckrich M, Joffe S, Lee SJ, LeMaistre CF, LeRademacher J, Loberiza F, Logan B, Parsons SK, Repaczki-Jones R, Robinett P, Rizzo JD, Murphy E, Denzen EM

National survey of hematopoietic cell transplantation center personnel, infrastructure, and models of care delivery

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1308-1314. doi:10.1016/ j.bbmt.2015.03.020. Epub 2015 Mar 31

PMC4466059

Clauser SB, Gayer C, Murphy E, Majhail NS, Baker KS

Patient centeredness and engagement in quality-of-care oncology research

Journal of Oncology Practice. 2015 May 1; 11(3):176-179. doi:10.1200/JOP.2015.003749. Epub 2015 Apr 7


Preussler JM, Mau L-W, Majhail NS, Bevans M, Clancy E, Messner C, Parran L, Pederson KA, Ferguson SS, Walters W, Murphy EA, Denzen EM

Patient housing barriers to hematopoietic cell transplantation: results from a mixed-methods study of transplant center social workers

Supportive Care in Cancer. doi:10.1007/ s00520-015-2872-9. Epub 2015 Aug 15


CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS

Page | 116

APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS

The following publications were generated by the Bioinformatics Research Program, which provides expertise in, and conducts research on, translational and operational bioinformatics. For more information, see Section 2.5.

BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID

Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP

Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems

Journal of Oncology Practice. 11(2):e120-e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1

PMC4371120

Slater N, Louzoun Y, Gragert L, Maiers M, Chatterjee A, Albrecht M

Power laws for heavy-tailed distributions: modeling allele and haplotype diversity for the National Marrow Donor Program

PLoS Computational Biology. 11(4):e1004204. doi:10.1371/journal.pcbi.1004204. Epub 2015 Apr 22

PMC4406525

Petz LD, Burnett JC, Li H, Li S, Tonai R, Bakalinskaya M, Shpall EJ, Armitage S, Kurtzberg J, Regan DM, Clark P, Querol S, Gutman JA, Spellman SR, Gragert L, Rossi JJ

Progress toward curing HIV infection with hematopoietic cell transplantation

Stem Cells and Cloning: Advances and Applications. 2015(8):109-116. doi:10.2147/SCCAA.S56050. Epub 2015 Jul 28

PMC4524463

Hollenbach JA, Saperstein A, Albrecht M, Vierra-Green C, Parham P, Norman PJ, Maiers M

Race, ethnicity and ancestry in unrelated transplant matching for the National Marrow Donor Program: a comparison of multiple forms of self-identification with genetics

PLoS One. doi:10.1371/journal.pone.0135960. Epub 2015 Aug 19

PMC4545604


Page | 117


Besse K, Maiers M, Confer D, Albrecht M

On modeling HLA-identical sibling match probability for alloHCT: estimating the need for an unrelated donor source

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.09.012. Epub 2015 Sep 5

N/A

Single RM, Strayer N, Thomson G, Paunic V, Albrecht M, Maiers M

Asymmetric linkage disequilibrium: tools for assessing multiallelic LD

Human Immunology. doi:10.1016/j.humimm.2015.09.001. Epub 2015 Sep 7

N/A

Mack SJ, Milius RP, Gifford BD, Sauter J, Hofmann J, Osoegawa K, Robinson J, Groeneweg M, Turenchalk GS, Adai A, Holcomb C, Rozemuller EH, Penning MT, Heuer ML, Wang C, Salit ML, Schmidt AH, Parham PR, Müller C, Hague T, Fischer G, Fernandez-Viña M, Hollenbach JA, Norman PJ, Maiers M

Minimum information for reporting next generation sequence genotyping (MIRING): guidelines for reporting HLA and KIR genotyping via next generation sequencing

Human Immunology. doi:10.1016/j.humimm.2015.09.011. Epub 2015 Sep 25


Magalon J, Maiers M, Kurtzberg J, Navarrete C, Rubinstein P, Brown C, Schramm C, Larghero J, Katsahian S, Chabannon C, Picard C, Platz A, Schmidt A, Katz G

Banking or bankrupting: strategies for sustaining the economic future of public cord blood banks

PLoS One. 10(12):e0143440. Epub 2015 Dec 1

PMC4666404


Page | 118


Buck K, Wadsworth K, Setterholm M, Maiers M, Confer D, Hartzman R, Schmidt A, Yang SY, Dehn J

7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.12.012. Epub 2015 Dec 24

N/A

CIBMTR 2015 Annual Report APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS

Page | 119

APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS

The following publications were generated by the Statistical Methodology Research Program, which develops and evaluates the statistical models used in HCT. For more information, see Section 2.6.

STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS


Keiding N, Andersen PK, Zhang MJ

Editorial: To the memory of John P. Klein

Lifetime Data Analysis. 2015 Apr 1; 21(2):157-159. doi:10.1007/ s10985-015-9320-5. Epub 2015 Feb 7

N/A

Wang T, He P, Ahn KW, Wang X, Ghosh S, Laud P

A re-formulation of generalized linear mixed models to fit family data in genetic association studies

Frontiers in Genetics. 6(120):1-10. doi:10.3389/ fgene.2015.00120. Epub 2015 Mar 31

PMC4379931

Eriksson F, Li J, Scheike T, Zhang M-J

The proportional odds cumulative incidence model for competing risks

Biometrics. 2015 Sep 1; 71(3):687-695. doi:10.1111/biom.12330. Epub 2015 May 26

PMC4608382

He P, Eriksson F, Scheike TH, Zhang M-J

A proportional hazards regression model for the subdistribution with covariates-adjusted censoring weight for competing risks data

Scandinavian Journal of Statistics, Theory and Applications. doi:10.1111/sjos.12167. Epub 2015 Jun 5


Brazauskas R, Logan BR Observational studies: matching or regression?

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.12.005. Epub 2015 Dec 19


CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS

Page | 120

APPENDIX D6: COORDINATING CENTER PUBLICATIONS

The following publications incorporated major contributions from the CIBMTR Coordinating Center and Scientific Directors.

COORDINATING CENTER PUBLICATIONS


Majhail NS, Giralt S, Bonagura A, Crawford S, Farnia S, Omel JL, Pasquini M, Saber W, LeMaistre CF

Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):583-588. doi:10.1016/ j.bbmt.2014.12.030. Epub 2015 Jan 29

N/A

Sucheston-Campbell LE, Clay A, McCarthy PL, Zhu Q, Preus L, Pasquini M, Onel K, Hahn T

Identification and utilization of donor and recipient genetic variants to predict survival after HCT: are we ready for primetime?

Current Hematologic Malignancy Reports. 2015 Mar 1; 10(1):45-58. doi:10.1007/ s11899-014-0246-x. Epub 2015 Feb 21

PMC4352187

Gratwohl A, Pasquini MC, Aljurf M, Atsuta Y, Baldomero H, Foeken L, Gratwohl M, Bouzas LF, Confer D, Frauendorfer K, Gluckman E, Greinix H, Horowitz M, Iida M, Lipton J, Madrigal A, Mohty M, Noel L, Novitzky N, Nunez J, Oudshoorn M, Passweg J, van Rood J, Szer J, Blume K, Appelbaum F, Kodera Y, Niederwieser D

One million haemopoietic stem-cell transplants: a retrospective observational study

The Lancet Haematology. 2(3):e91-e100. doi:10.1016/S2352-3026(15)00028-9. Epub 2015 Mar 1

N/A


Page | 121

COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID

Shah N, Callander N, Ganguly S, Gul Z, Hamadani M, Costa L, Sengsayadeth S, Abidi M, Hari P, Mohty M, Chen Y-B, Koreth J, Landau H, Lazarus H, Leather H, Majhail N, Nath R, Osman K, Perales M-A, Schriber J, Shaughnessy P, Vesole D, Vij R, Wingard J, Giralt S, Savani BN

Hematopoietic stem cell transplantation for multiple myeloma: guidelines from the American Society for Blood and Marrow Transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1155-1166. doi:10.1016/j.bbmt. 2015.03.002. Epub 2015 Mar 11


Eapen M Hematopoietic cell transplantation for acute leukemia: selecting donors

Haematologica. 2015 Apr 1; 100(4):414-415. doi:10.3324/haematol. 2015.124974. Epub 2015 Apr 1

PMC4380712

Khera N From evidence to clinical practice in blood and marrow transplantation

Blood Reviews. doi:10.1016/j.blre. 2015.04.001. Epub 2015 Apr 9

PMC4610823

Graff TM, Singavi AK, Schmidt W, Eastwood D, Drobyski WR, Horowitz M, Palmer J, Pasquini M, Rizzo DJ, Saber W, Hari P, Fenske TS

Safety of outpatient autologous hematopoietic cell transplantation for multiple myeloma and lymphoma

Bone Marrow Transplantation. 2015 Jul 1; 50(7):947-953. doi:10.1038/ bmt.2015.46. Epub 2015 Apr 13

PMC4490016

Martin PJ, Lee SJ, Przepiorka D, Horowitz MM, Koreth J, Vogelsang GB, Walker I, Carpenter PA, Griffith LM, Akpek G, Mohty M, Wolff D, Pavletic SZ, Cutler CS

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1343-1359. doi:10.1016/ j.bbmt.2015.05.004. Epub 2015 May 15

PMC4506719


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Gale RP, Eapen M Who is the best alternative allotransplant donor?

Bone Marrow Transplantation. 2015 Jun 1; 50(S2):S40-S42. doi:10.1038/ bmt.2015.94. Epub 2015 Jun 4

PMC4520408

Renner R, Carlis J, Maiers M, Rizzo JD, O'Neill C, Horowitz M, Gee K, Confer D

Integration of hematopoietic cell transplantation outcomes data: data standards are not enough

Data Integration in the Life Sciences. 2015 Jul 8; 9162:139-146. doi:10.1007/978-3-319-21843-4_11

N/A

Booth GS, Gehrie EA, Jagasia MH, Shaw BE, Savani BN

When can you discard stem cells?

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2015.07.024. Epub 2015 Aug 4

N/A

Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Savani BN, Veys PA, Bredeson CN, Giralt SA, LeMaistre CF

Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2015.07.032. Epub 2015 Aug 7

N/A

Deeg HJ, Bredeson C, Farnia S, Ballen K, Gupta V, Mesa RA, Popat U, Hari P, Saber W, Seftel M, Tamari R, Petersdorf EW

Hematopoietic cell transplantation as curative therapy for patients with myelofibrosis: long-term success in all age groups

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Nov 1; 21(11):1883-1887. doi:10.1016/ j.bbmt.2015.09.005. Epub 2015 Sep 11

PMC4604067


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Pagliuca S, Gérard L, Kulasekararaj A, Eapen M, Boutboul D, Martin H, Salvino MA, Knol-Bout C, Dufour C, Peffault de Latour R, Marsh J

Characteristics and outcomes of aplastic anemia in HIV patients: a brief report from the severe aplastic anemia working party of the European Society of Blood and Bone Marrow Transplantation

Bone Marrow Transplantation. doi:10.1038/ bmt.2015.252. Epub 2015 Oct 19

N/A

CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS

Page | 124

APPENDIX E: PRESENTATIONS

2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI

BMT CTN 0201

5 year results of BMT CTN 0201: unrelated donor bone marrow is associated with better psychological well-being and less burdensome chronic graft-versus-host disease symptoms than peripheral blood * Selected as a 2016 Highlight of ASH

Oral SJ Lee

BMT CTN 0601

A multicenter Phase II trial of unrelated donor reduced intensity bone marrow transplantation for children with severe sickle cell disease (SCURT): Results of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0601) Study

Oral S Shenoy

BMT CTN 0805

Multicenter US intergroup study of intensive chemotherapy plus dasatinib followed by allogeneic stem cell transplant in patients with Philadelphia chromosome positive acute lymphoblastic leukemia younger than 60 * Late Breaking Abstract

Oral F Ravandi

BMT CTN 0901

Results of a Phase III randomized, multi-center study of allogeneic stem cell transplantation after high vs reduced intensity conditioning in patients with myelodysplastic syndrome or acute myeloid leukemia * Late Breaking Abstract

Oral BL Scott

CK11-02 A prognostic system predictive of outcomes in persons undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome

Oral B Shaffer

CK14-01 Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes: a Center for International Blood and Marrow Transplant Registry study

Poster A Gerds

GV12-01 Outcomes of grades II-IV acute graft-versus-host disease post allogeneic hematopoietic stem cell transplantation: How much progress was achieved?

Poster H Khoury

GV12-02

Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value when present in isolation or in addition to grade I or other grade II-defining GvHD manifestations

Oral S Nikiforow


Page | 125


HS13-01 The impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation

Oral A El-Jawahri

HS13-02 A study of predictors of clinical outcomes and healthcare utilization in children with sickle cell disease undergoing allogeneic hematopoietic cell transplantation

Oral S Arnold

IB11-01

Evaluation of the impact of non-inherited maternal antigens on the outcome of HLA mismatched unrelated donor hematopoietic stem cell transplantation for hematological malignancies on behalf of the ALWP of the EBMT and the CIBMTR

Poster J Pingel

IB12-03 Investigating effect of genetic admixture and donor / recipient genetic disparity on transplant outcomes Poster A Madbouly

LK13-04

Comparison of post allogeneic hematopoietic cell transplantation outcomes after matched related donor versus matched unrelated donor HCT in adults with acute lymphoblastic leukemia

Poster E Segal

LK14-02 Outcomes of allogeneic transplantation in patients aged ≤ 60 years with acute myeloid leukemia in second complete remission: a CIBMTR cohort analysis

Poster F Michelis

LK14-03 Autologous transplant, and not ATO alone, remains the preferred therapy for relapsed APL: a report from the CIBMTR, EBMT, and two specialized centers

Oral M Tallman

LY06-03

Allogeneic stem cell transplantation for relapsed / refractory follicular lymphoma: a joint study between the European Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research

Oral A Sureda

LY14-02

Reduced-intensity allogeneic hematopoietic cell transplantation provides durable progression-free survival in a subset of diffuse large B-cell lymphoma patients relapsing after autologous HCT

Oral T Fenske


Page | 126


LY15-01

Survival after T-cell replete haploidentical related donor transplant using post-transplant cyclophosphamide compared with matched unrelated donor transplant for lymphoid malignancies * Selected as a 2016 Highlight of ASH

Oral M Kharafan-Dabaja

MM13-02 Autologous hematopoietic cell transplantation in patients with high risk multiple myeloma: post-transplant responses do not translate to longer survival

Poster M Sharma

MM15-01 Post transplant therapy is more important than induction regimen choice in autologous hematopoietic cell transplantation recipients for multiple myeloma

Oral R Cornell

RCI BMT 09PLEX

A Phase II study evaluating the safety and efficacy of subcutaneous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematologic malignancies

Oral S Devine

RT09-04 / IB09-06

Combined donor and recipient non-HLA genotypes show evidence of genome-wide association with transplant related mortality after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study)

Oral L Sucheston-Campbell

RT09-04 / IB09-06

Genome wide association study of overall and progression-free survival after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study)

Oral T Hahn

RT09-04c

Evidence for heterogeneous genetic associations with acute lymphoblastic leukemia by cytogenetics and sex in high risk patients treated with matched unrelated donor allogeneic blood or marrow transplant

Poster A Clay

SC11-06 Outcome of patients 65 years and older with myelodysplastic syndrome receiving allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age

Oral E Atallah


Page | 127

2015 BMT TANDEM MEETINGS

Study Title Type Principal Investigator

Bio- informatics Metadata / BRIDG integration process Oral J Pollack

Bio- informatics Stem cell transplant interoperability using BRIDG Oral RP Milius

BMT CTN More infections with transplantation of bone marrow versus peripheral-blood stem cells from unrelated donors Oral J Young

BMT CTN 0302 / 0802

Prognostic impact of follistatin in acute graft-versus-host disease: results from BMT CTN 0302 and 0802 Oral S Holtan

CK13-02 Allogeneic hematopoietic cell transplantation for adult chronic myelomonocytic leukemia Oral H Duong

DS05-02b & RCI BMT

06-DON

Health-related quality of life among older adult related hematopoietic stem cells donors (>60 yrs.) is equivalent to or better than that of younger adult related donors (18-60 yrs.)

Oral G Switzer

DS05-02c / RCI BMT 06-DON

Acute toxicities of related adult donors compared to unrelated adult Oral M Pulsipher

DS09-04

The effect of race, socioeconomic status, and collection center size on bone marrow and peripheral blood stem cell donor experiences at National Marrow Donor Program collection centers

Oral M Pulsipher

HS13-03 Clinical outcomes among unrelated donor transplant recipients for acute myelogenous leukemia as a function of socioeconomic status and related transcriptome differences

Oral J Knight

HSR 13-01 Administrative claims data for cost analyses in hematopoietic cell transplantation: the good, the bad and the ugly Poster J Preussler

HSR 13-02 Patient and provider preferences for survivorship care plans for allogenic hematopoietic cell transplantation survivors: a qualitative study

Poster E Murphy

HSR 14-02 Hematopoietic cell transplantation multidisciplinary care teams: burnout, moral distress and career satisfaction Poster J Neumann


Page | 128

2015 BMT TANDEM MEETINGS


IB13-05

MHC class I chain-related gene A (MICA) donor-recipient mismatches and MICA-129 polymorphism in unrelated donor hematopoietic stem cell transplants for hematological malignancies: a CIBMTR study

Poster M Askar

IS13-01

Impact of race on graft-versus-host disease rates after HLA-matched sibling bone marrow or peripheral blood hematopoietic cell transplantation: Comparison of North American Caucasian versus Japanese populations

Oral J Kanda

LK13-03 Allogeneic transplant for acute biphenotypic leukemia: characteristics and outcome in the CIBMTR database Oral R Munker

LY12-01 Positive pre-allogeneic hematopoietic cell transplantation PET scan in patients with non-Hodgkin lymphoma predicts higher risk of relapse but has no impact on survival

Oral V Bachanova

LY13-02

Risk factors predicting outcomes of autologous hematopoietic cell transplantation in children, adolescents and young adults with relapsed / refractory classical Hodgkin lymphoma: a CIBMTR analysis

Oral P Satwani

2015 AMERICAN SOCIETY OF CLINICAL ONCOLOGY ANNUAL MEETING


LY13-03

Reduced intensity conditioning allograft as first transplant approach in relapsed / refractory grade III follicular lymphoma is associated with improved outcomes in long-term survivors

Oral E Klyuchnikov

BMT CTN 0704

Updated analysis of CALGB / ECOG / BMT CTN 100104: Lenalidomide vs. placebo maintenance therapy after single autologous stem cell transplant for multiple myeloma

Poster SA Holstein


Page | 129

2015 EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION (EBMT) ANNUAL MEETING


Bio-informatics

HML 1.0

Reporting NGS-based HLA & KIR genotyping using MIRING principles Oral RP Milius

DS05-02f / RCI BMT 06-DON

Health-related quality of life among pediatric hematopoietic stem cell donors Oral G Switzer

DS14-01 (US)

Significant improvements in the practice patterns of related donor care in US transplant centres Poster C Anthias

DS14-01 (EBMT)

JACIE accreditation significantly improves compliance with international recommendations for related donor care in EBMT transplant centres

Poster C Anthias

IB12-02b Uni-directional and bi-directional non-permissive HLA-DPB1 T cell epitope group mismatches have similar risk associations in 10/10 matched unrelated donor HCT

Oral K Fleischhauer

IB12-04b Effect of HLA-C allele matching in the context of patients HLA-C encoded KIR ligand grouping (C1 or C2) on outcomes of unrelated hematopoietic stem cell transplantation

Oral J Fischer

IB12-06

KIR B genotype in HLA-matched unrelated donor protects from relapse and improves progression-free survival after allogeneic transplantation for relapsed / refractory non-Hodgkin lymphoma (This presentation was withdrawn because the PI was unable to attend the meeting)

Poster V Bachanova

2015 EUROPEAN HEMATOLOGY ASSOCIATION CONGRESS


LY13-03 Reduced intensity conditioning allo transplantation is associated with superior long-term disease control in relapsed / refractory grade I/II follicular lymphoma

Oral E Klyuchnikov

BMT CTN 0102

Heavy light chain ratio normalization allows identification of electrophoretic non-complete response patients with improved outcomes: a long term follow up update for BMT CTN 0102 correlative study

Poster A D’Souza


Page | 130

2015 EUROPEAN IMMUNOGENETICS AND HISTOCOMPATIBILITY CONFERENCE


Bio-informatics Using SNPs to improve phasing of HLA haplotypes Oral V Paunic

Bio-informatics Impudigree: an imputation-based automated pedigree tool Oral J Freeman

Bio-informatics

Improved HLA-based race / ethnic classification using donor geography and census demographic data Poster L Gragert

Bio-informatics HLA diversity in the Ezer Mizion Registry Poster M Halagan

Bio-informatics

HLA class II six-locus DRB3/4/5~DRB1~DQA1~DQB1~DPA1~ DPB1 high resolution haplotype frequencies of the major US populations

Poster M Halagan

Bio-informatics

HLA allele and haplotype frequencies for Christian and Muslim Arab donors in Hadassah Registry Poster A Bishara

2015 INSTITUTE FOR HEALTHCARE ADVANCEMENT’S ANNUAL HEALTH LITERACY CONFERENCE


BMT CTN 1205

Easy-to-read informed consent forms for multi-center hematopoietic cell transplant clinical trials Poster H Moore

2015 INTERNATIONAL CONFERENCE ON LONG-TERM COMPLICATIONS OF TREATMENT OF CHILDREN AND ADOLESCENTS FOR CANCER


LE13-04 Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Poster M Ehrhardt


Page | 131

2015 PEDIATRIC BLOOD AND MARROW TRANSPLANT CONSORTIUM ANNUAL MEETING


LE13-04 Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Oral M Ehrhardt

2015 WORLD CONGRESS OF PSYCHO-ONCOLOGY (hosted by the International Psycho-Oncology Society and

American Psychosocial Oncology Society)


BMT CTN 0902

Cancer and treatment distress measurement over time in a multicenter cohort of hematopoietic cell transplantation recipients

Oral K Syrjala

BMT CTN 0902

Pre-transplant health-related quality of life factors as predictors of outcomes following hematopoietic cell transplantation

Oral J Knight

2015 WORLD MARROW DONOR ASSOCIATION MEETING


Bio-informatics

Key driver analysis of HLA diversity: analytically focused recruitment strategies for improving registry quality Poster A

Sivasankaran

Bio-informatics

Enhancing HML for electronic reporting of NGS-based HLA and KIR genotyping results Poster J Kathryn

CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS

Page | 132

APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS

This study development and management process pertains to studies for which the CIBMTR provides data and statistical support. Data sets are also made available to investigators who have their own statistical resources. Final analyses and manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission.

STUDY DEVELOPMENT AND MANAGEMENT PROCESS

Planned Protocol pending. Proposals remain in this preliminary stage until the PI creates a draft protocol.

In Progress

Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it.

Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee.

Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome.

Supplemental forms / data collection. Most studies use routinely-collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on transplant centers and supplement forms reimbursement costs.


Page | 133


In Progress (continued)

Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow-up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS-level statistician, sometimes working with the Clinical Research Coordinator, to ensure data quality: • Verifying selection criteria • Including and excluding patients so that the investigators can determine

whether the final study population is representative of the target population • Assessing follow-up • Determining the extent and nature of missing values and their potential effect

on the study • Resolving and reconciling data discrepancies / outliers by examining data

collection forms and communicating with centers and the PI

Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Coordinating Center personnel present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript.

Ongoing. A study in ongoing status is long-term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year.

Preliminary Results

Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. The Working Committee leadership review and revise the document, ensuring that the description and interpretation of the statistical analyses are accurate and contribute to the fundamental message of the manuscript. The Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript.


Page | 134


Preliminary Results

(continued)

Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician forwards all editor and reviewer comments to the PI and Statistical Director. The PI is expected to prepare a response, working with Coordinating Center staff who provide additional analyses of data, as needed. Coordinating Center personnel communicate with the journal, including re-submissions, in most cases.

In press. A publication is in press when it has been approved but does not yet have a citation.

Completed Published. A manuscript is considered published when a citation is available, including a PMCID number, if applicable. For a list of 2015 publications, see Appendix D.

CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS

Page | 135

APPENDIX G: CLINICAL TRIALS

Through the Clinical Trials Support Program, the Coordinating Center supports clinical trial planning and interpretation; data collection, including long-term follow-up data; and real-time accrual assessment. See Section 2.3 for more information.

APPENDIX G1: BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT

The BMT CTN (Section 2.3.1) is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation. A status of BMT CTN trials open for enrollment is included in this appendix and is available on the BMT CTN website. For additional information on completed Network trials, see the annual progress report on the BMT CTN website.

BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT

Protocol Number Title Status to Date

BMT CTN 1101

Phase III study comparing HLA-haploidentical related donor bone marrow versus double umbilical cord blood with reduced-intensity conditioning for patients with hematologic malignancies

• Opened to accrual Jun 2012 • 219 of 410 patients enrolled • Anticipated accrual completion in

2017

BMT CTN 1102

A multi-center biologic assignment trial comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome

• Opened to accrual Dec 2013 • 147 of 338 patients enrolled • Anticipated accrual completion in

early 2018

BMT CTN 1202

Prospective multi-center cohort for the evaluation of biomarkers predicting risk of complications and mortality following allogeneic HCT

• Opened to accrual Jun 2013 • 1,708 of 1,500 patients enrolled;

accrual target increased to enhance pediatric and minority patient accrual

• Anticipated accrual completion in Jun 2016

BMT CTN 1203

A multi-center Phase II trial randomizing novel approaches for graft-versus-host disease prevention compared to contemporary controls

• Opened to accrual Sep 2014 • 209 of 270 patients enrolled • Anticipated accrual completion in

April 2016

https://web.emmes.com/study/bmt2/public/NewProtocol.html


CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS

Page | 136

BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Number Title Status to Date

BMT CTN 1205

Easy-to-read informed consent (ETRIC) for hematopoietic cell transplantation clinical trials

• Opened to accrual Nov 2013 • 138 of 160 patients enrolled,

although accrual target may be increased

• Anticipated accrual completion in 2016

BMT CTN 1301

Phase III trial of calcineurin inhibitor-free interventions for prevention of graft-versus host-disease

• Opened to accrual Aug 2015 • 9 of 345 patients enrolled • Anticipated accrual completion in

early 2019

BMT CTN 1302

Phase II, double-blind Placebo controlled trial of maintenance ixazomib after allogeneic HCT for high risk multiple myeloma

• Opened to accrual Aug 2015 • 6 of 138 patients enrolled • Anticipated accrual completion in

June 2018

BMT CTN 1304

(DFCI 10-106)

A randomized Phase III study comparing conventional dose treatment using a combination of lenalidomide, bortezomib, and dexamethasone (RVD) to high-dose treatment with peripheral stem cell transplant in the initial management of myeloma in patients up to 65 years of age

• Opened to accrual by BMT CTN Nov 2013

• 514 of 660 patients enrolled • Anticipated accrual completion in

2016

BMT CTN 1505

(UT RECRUIT)

Randomized minority patient recruitment intervention trial

• Opened to accrual by BMT CTN May 2015

• Patient enrollment: N/A, BMT CTN centers randomized to intervention and control arms; center staff are research subjects

• Anticipated accrual completion in May 2017

CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS

Page | 137

APPENDIX G2: RCI BMT CLINICAL TRIALS

The RCI BMT (Section 2.3.2) provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting into such groups as the BMT CTN or the national cancer cooperative groups. A status of its projects is included in this appendix.

RCI BMT CLINICAL TRIALS Protocol # Title Status To Date

Astellas

Unrelated donor sample collection for donors whose recipient is enrolled in a randomized, double-blind, placebo-controlled, Phase III trial to evaluate the protective efficacy and safety of a therapeutic vaccine, ASP0113, in cytomegalovirus-seropositive recipients undergoing allogeneic hematopoietic cell transplant

• Donor sample collection only • Opened to accrual Apr 2014 • Two donors enrolled • RCI BMT closed study Dec 2014

12-MOXE

Study of the anti-CD22 recombinant immunotoxin moxetumomab pasudotox (CAT-8015, HA 22) in children with B-lineage acute lymphoblastic leukemia and minimal residual disease prior to allogeneic hematopoietic cell transplantation

• Opened to accrual May 2015 • One patient enrolled • Study closed Sep 2015 due to

pharmaceutical company decision

09-SQOL Pilot study to assess the feasibility of collecting quality of life data in collaboration with the Stem Cell Therapeutic Outcomes Database

• Closed to accrual Sep 2013 • 301 adults and 89 pediatric

recipients enrolled • Final subject follow-up expected

by end of 2014 • Data analysis in process

11-TREO Multi-center study evaluating treosulfan, fludarabine, and low-dose TBI in children with AML / MDS undergoing allogeneic HCT

• Closed to accrual April 2014, about one year earlier than expected

• 40 of 40 targeted enrollment • Follow-up continues • Abstract submitted to 2016

EBMT Annual Meeting

COG-KIR A multi-center study examining donor NK-cell receptors and patient outcomes

• COG closed accrual May 2014 • URD sample management • 609 of 1,200 samples collected • 145 out of 400 targeted

enrollment


Page | 138


DS05-02, 06-DON

RDSafe: A multi-institutional study of hematopoietic stem cell donor safety and quality of life

• Closed to accrual July 2014 • 1,812 donors enrolled • Follow up assessments

completed July 2015 • Abstract presented at 2015 BMT

Tandem Meetings • Data review and analysis as well

as manuscript planning in progress

PBMTC ONC 1001 / 09-

MRD

A multi-center study to determine the role of minimal residual disease testing before and after HCT for pediatric acute myeloid leukemia

• Closed to accrual Oct 2014 • 150 total enrollment • Follow-up continues

09-PLEX

A phase II study evaluating the safety and efficacy of intravenous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematological malignancies

• Closed to accrual Dec 2014 • Follow-up completed Feb 2016 • Oral abstract presented at 2015

ASH Annual Meeting • Data review and analysis as well

as manuscript planning in process

rHuG-CSF

Long-term follow-up study evaluating hematologic and non-hematologic cancers, thrombotic events, and autoimmune disorders in unrelated donors undergoing bone marrow harvest versus peripheral blood stem cell mobilization with recombinant human granulocyte colony-stimulating factor

• Closed to accrual Oct 2015 • 21,569 total enrollment • Follow-up continues through

2020

PBSC Filgrastim-mobilized peripheral blood stem cells for allogeneic transplantation with unrelated donors

• Opened to accrual Apr 1996 • Just under 25,000 URDs enrolled • Will close to accrual upon FDA

license; another protocol will open for all unlicensed product

10-CBA

A multi-center access and distribution protocol for unlicensed cryopreserved cord blood units for transplantation in pediatric and adult patients with hematologic malignancies and other indications

• Opened to accrual Oct 2010 • 2,757 enrolled • Open indefinitely to allow

distribution and access to unlicensed cord units

10 CMS-MDS-1

Assessment of allogeneic HCT in Medicare beneficiaries with MDS and related disorders

• Opened to accrual Dec 2010 • 1,294 patients enrolled


Page | 139


KIR-DS A multi-center study looking at the selection of a favorable KIR donor

• Opened to accrual Jun 2011 • URD sample management • 1,942 samples facilitated • Amendment in process to alter

donor sample logistics, which will eliminated CIBMTR staff involvement

Statin Impact of donor statin use on graft-versus-host disease after unrelated donor HCT; URD data collection

• Opened to accrual Oct 2011 • 5,569 of 7,000 targeted donors

enrolled

BMT CTN 1102-QOL

A study comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome

• Survey Research Group performing QOL assessments

• 210 total assessments completed

13-TLEC Prospective non-therapeutic study, assessing the long-term toxicity of HCT for childhood leukemia

• Opened to accrual Mar 2015 • 53 of 340 patients enrolled

13-SCP

A randomized study to evaluate the impact of survivorship care planning on cancer survivors self-management and adherence to care recommendations and utilization of follow-up care

• In collaboration with Health Services Research Program

• Opened to accrual Apr 2015 • 181 of 495 patients enrolled

BMT CTN 1102-

Ancillary CEA study

A cost effectiveness ancillary study to the parent study 1102 above

• Collaborating with Fred Hutchinson Cancer Research center to perform Cost Effectiveness Analysis (CEA) study

• Survey Research Group to perform CEA survey collection

• First subject contacted in Oct 2015

HPD High priority donor project supporting Be The Match Operations

• First team meeting held in June 2015

• First subjects contacts by the Survey Research Group in Sep 2015

15-MMUD Study of transplantation of HLA-mismatched unrelated donor bone marrow for patients with hematologic malignancies

• Protocol team established Oct 2015

• Protocol development in process

CIBMTR 2015 Annual Report APPENDIX H: FORMS SUBMISSION PROCESS

Page | 140

APPENDIX H: FORMS SUBMISSION PROCESS

• Center submits CRID Assignment Form (Form 2804), and CRID is generated • Indication for CRID Assignment Form (Form 2814) is added to Forms Due list • Center completes Indication Form and reports indication as HCT • Pre-TED (Form 2400) is added to Forms Due list • Center completes and submits Pre-TED • Pre-TED data are processed through the selection algorithm resulting in CRF or TED track

o If autologous recipient declines consent for research, stop here. Otherwise, follow the appropriate track below

CRF Track TED Track

1 Forms 2004, 2005, and 2006 are added, depending on donor type and if the donor has been used for a prior transplant.*

Forms 2004, 2005, and 2006 are added, depending on donor type, consent for sample repository, and if the donor has been used for a prior transplant.*

2 Baseline form 2000, disease specific inserts, and Follow-up Forms are added to Forms Due list.

Post-TED Follow-up Form 2450 is added to Forms Due list.

3 Center completes Baseline form after infusion. Center completes designated Post-TED Forms at appropriate time points.

4 Center completes designated CRF Follow-up Forms at appropriate time points.

Is recipient alive? If yes, go to Step 5. If no, report the death on the follow-up form, and go to Reporting Recipient Death.

5 Is recipient alive? If yes, go to Step 6. If no, report the death on the follow-up form, and go to Reporting Recipient Death.

Did recipient have a subsequent transplant? If yes, go to Step 6. If no, continue reporting at next time point (Step 3).

6 Did recipient have subsequent transplant? If yes, go to Step 7. If no, continue reporting at next time point (Step 4).

Subsequent transplant is reported on the next available follow-up form.

7 When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.

When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.

8 Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.

Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.

Reporting Recipient Death

Death Form 2900 is completed to report the recipient’s death.**

The recipient’s death is reported on the Post TED. A 2900 Death Form should not be completed for patients on the TED track.

* For more details regarding when Forms 2004, 2005, and 2006 are required, see “How Forms Come Due (2004, 2005, and 2006)”.

**Complete Death Form 2900 even if autopsy is pending. Another death form will be requested to confirm cause of death if autopsy was pending.

CIBMTR 2015 Annual Report APPENDIX I: WEBSITES

Page | 141

APPENDIX I: WEBSITES

Throughout this report, electronic links to webpages and documents are provided. If you are unable to access items using the links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). URLs for the websites mentioned in this report are provided here.

Name URL

Be The Match bethematch.org Be The Match Clinical bethematchclinical.org BMT CTN bmtctn.net CIBMTR cibmtr.org CIBMTR Collaborate collaborate.cibmtr.org CIBMTR Portal portal.cibmtr.org HRSA Blood Cell Transplant bloodcell.transplant.hrsa.gov

CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY

Page | 142

APPENDIX J: GLOSSARY

Abbreviation/ Acronym Meaning

AGNIS A Growable Network Information System alloHCT allogeneic hematopoietic cell transplantation AML acute myeloid (myelogenous) leukemia ASBMT American Society of Blood and Marrow Transplantation ASH American Society of Hematology autoHCT autologous hematopoietic cell transplantation BMT bone marrow transplant BMT CTN Blood and Marrow Transplant Clinical Trials Network BRIDG Biomedical Research Integrated Domain Group CALGB Cancer and Leukemia Group B (member Alliance for Clinical Trials in Oncology) CED Coverage with Evidence Determination CIBMTR Center for International Blood and Marrow Transplant Research CME continuing medical education CMS Centers for Medicare and Medicaid Services COG Children’s Oncology Group CPI Continuous Process Improvement CRF Comprehensive Report Form CRID CIBMTR Recipient Identification Number DBtC Data Back to Centers application DFCI Dana Farber Cancer Institute DISCO Data and Information for Statistical Center Operations DRI Disease Risk Index EBMT European Group for Blood and Marrow Transplantation eDBtC enhanced Data Back to Centers application FACT Foundation for the Accreditation of Cellular Therapy FDA Food and Drug Administration HCT hematopoietic cell transplantation HIPAA Health Insurance Portability and Accountability Act HIV human immunodeficiency virus HLA human leukocyte antigen HRSA Health Resources and Services Administration IND Investigational new drug IRB Institutional Review Board IT Information Technology KIR killer-cell immunoglobulin-like receptors MCW Medical College of Wisconsin MDS myelodysplastic syndrome MED-A, MED-B Minimum Essential Data forms N/A not applicable NCI National Cancer Institute

CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY

Page | 143

Abbreviation/ Acronym Meaning

NK natural killer (cell) NHLBI National Heart, Lung, and Blood Institute NIAID National Institute of Allergy and Infectious Disease NIH National Institutes of Health NMDP National Marrow Donor Program PBMTC Pediatric Blood and Marrow Transplant Consortium PBSC peripheral blood stem cell PI principal investigator PMCID PubMed Central unique identifier QOL quality of life RCI BMT Resource for Clinical Investigations in Blood and Marrow Transplant RDSafe Related Donor Safety Study RITN Radiation Injury Treatment Network SCTOD Stem Cell Therapeutic Outcomes Database TBD to be determined TED Transplant Essential Data URD unrelated donor US United States vs versus

Milwaukee CampusMedical College of Wisconsin 9200 W. Wisconsin Ave., Suite C5500Milwaukee, WI 53226 USA(414) 805-0700cibmtr.org

Minneapolis CampusNational Marrow Donor Program/ Be The Match 500 N 5th StreetMinneapolis, MN 55401 USA(612) 627-5800cibmtr.org

© 2016 The Medical College of Wisconsin, Inc. and the National Marrow Donor Program

CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and Medical College of Wisconsin

The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID).

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