Dao-Pei LuMay 2010, Shanghai

Hematopoietic Stem Cell

Transplantation (HSCT) in

China (2010)

1CSBMT and Syn-HSCT

Establishment of CSBMT(Chinese Society of BMT)

20072007

20052005

20092009

Hangzhou, establishment of Chinese BMT Cooperation Group in APBMT and China BMT Group meeting

Beijing, establishment of CSBMT in ISH-APD & APBMT meeting

Hong-Kong, Chinese BMT Forum

1st CSBMT Conference (2009, Hong Kong)

1st CSBMT Conference (2009, Hong Kong)

CSBMT Newsletter

Syn-HSCT in Chinese--A report of 94 cases from 32

centers in CSBMT

Patient characteristics

CSBMT

AA

AML

large granular lymphocytosis

Neuroblastoma

lymphoma

MDS

CML

ALL n=94

OS of Syn-HSCT (n=94)

DFS of Syn-HSCT (n=94)

Follow-up the longest survivor of HSCT in the world

2Overview of HSCT in

China

HSCT Number in Different Cities in China

HSCT Number in Different Units in China

HSCT indications in China（ 2009 ）

A ML

A L LC ML

MDS

L ym phoidm alig nancy

MM

Other leukemia

Autoimmune diseaseSolid tumor

AA

Types of HSCT in China（ 2009 ）

Identicalsibling

Haplo

Unrelated

Auto

Syngeneic

GIAC Principle

Combined BM & PBC:

ATG A:

Immuno-suppression intensified and prolonged

I :

G-CSF mobilizationGG:

Haploidentical HSCT(Haplo-HSCT)

Lu et al. Blood 2006; 107: 3065

Haploidentical Blood and Marrow Transplantation in

Hematological Malignancies: A Single-

Center Report of 290 patients

Tong Wu, Dao-Pei Lu, et al.

Beijing Daopei Hospital, Beijing, China

August 2009

14th APBMT

Outstanding Oral Presentation Award

(Haplo-HSCT)

14th APBMT (August 2009, Korea)

20 40 60 80 1000

20

40

60

80

100II-IV aGVHD

III-IV aGVHD

Days after BMT

Per

cen

t

34.9%

16.0%

Acute GVHD of Haplo-HSCT (Beijing Daopei Hospital, n=290)

1 2 3 4 5 6 70

20

40

60

80

100standard risk

high risk

advanced

Years after BMT

LF

S

73.9%

51.0%

22.2%

P<0.0001

LFS of Haplo-HSCT (Beijing Daopei Hospital, n=290)

1 2 3 4 5 6 70

20

40

60

80

100standard risk

high risk

advanced

Years after BMT

OS

77.3%

58.1%

29.5%

P<0.0001

OS of Haplo-HSCT (Beijing Daopei Hospital, n=290)

Indications of Unrelated-HSCT (U-HSCT)

（ Beijing Daopei Hospital, n=182)

Acute GVHD of U-HSCT (Beijing Daopei Hospital, n=182)

5yr-OS of U-HSCT (Beijing Daopei Hospital, n=182)

72.2%

5yr-DFS of U-HSCT (Beijing Daopei Hospital, n=182)

64.9%

U-HSCT with Haplo-BM (Beijing Daopei Hospital, n=182)

Successful Rescue of Refractory/Recurrent

Myelogenous Leukemia by Allogeneic HSCT and

Prophylactic Immunotherapy

Beijing & Shanghai Daopei Hospitals

Refractory/recurrent myelogenous leukemia by HSCT

and immunotherapy• 30 cases (AML 29 ， CML-BC myeloid 1)

• Median blasts in BM 36 ( 20-87 )%

• Types of HSCT:

– Matched sibling 5

– Unrelated 7

– Haploidentical 18

• Median follow-up 17 (5 － 37) months

Conditioning• <40% blasts in BM

– Ara-C + BuCy 13

• >40% blasts in BM

– Chemotherapy + reduced BuCy 6

• Impaired organs：– Fludarabine substituted for Cy 11

Prophylaxis of Relapse

–Immunosuppressants were tapered off early post-HSCT

–Prophylactic immunotherapy if no GVHD

•Cellular: DLI, DC-CIK, NK cells

•Humoral: IL-2, IFN-a, thymosin

Refractory/recurrent myelogenous leukemia by HSCT

and immunotherapy

OS

EFS

3 Further application of

HSCT

A Special Neonatal Case of SCID cured by U-CBT

Dao-pei Hospital & Pediatric Hospital, Fu Dan University

Reconstitution

PLT

WBCN#L#

U-CBT 前

U-CBT 后 5 个月

Familial Hemophagocytic Lymphohistiocytosis (FHL)

treated by U-HSCT

Beijing Daopei Hospital

Patient Zhang x. 13 yr girl

• “Lymphoma” was diagnosed and treated in other hospitals.

• FHL was diagnosed on admission.

Zhang’s Family Tree

Diagnosed as FHL-2

MACPF 结 构 域 野 生 型 PRF1-168S

MACPF 结 构 域 突 变 型 PRF1-168N

MHD2 结构域 野生型 UNC13D-863G MHD2 结构域 突变型 UNC13D-863D

Protein conformation of PRF1 gene mutations

Paternal

Maternal

Perforin normalization post HSCT of Zhang Y.

-3 -2 -1 1 32 4

Month

--3 -2 --1 1 32 4

Autologous cord blood transplantation (Auto-

CBT) in children

Beijing Children’s Hospital (BCH)

Beijing Daopei Hospital (BDH)

Auto-CBT in ChildrenDate

of HSCT

Hospital Diagnosis Follow-up

(month)

Outcome

2009.4 BCH Neuroblastoma

2 CR

2010.1 BDH SAA 4 CR

2010.2 BDH SAA 3 CR

Auto-CBT for pediatric SAABeijing Daopei Hospital

Before HSCT

Summary

1 、 CSBMT must play an important role in HSCT continuously.

2 、 Large series of syn-HSCT show excellent outcome in both nonmalignant and malignant hematological diseases.

Summary3 、 HSCT in China has been developing

rapidly. The outcomes of Haplo-HSCT and U-HSCT have achieved top level in the world in experienced units.

4 、 DFS in refractory/recurrent myeloid leukemia treated by allo-HSCT and immunotherapy has been improved significantly.

Summary

5 、 More and more diseases could be treated by HSCT, such as allo-HSCT for inherited diseases (SCID, FHL) and auto-CBT for solid tumor and SAA.

Acknowledgment