19-21 November 2021 - tifeducation.org

Organizer With the support of

19-21 November 2021

15th International Conference

on Thalassaemia & Other Haemoglobinopathies

17th TIF Conference for Patients & Parents

&

www.tifeducation.org

virtual

#TIF2021

ConferenceProgramme

http://www.tifeducation.org

315th International Conference on Thalassaemia & Other Haemoglobinopathies

& 17th TIF Conference for Patients & Parents

Welcome Message

Dear Friends,

We are priviledged to welcome you to the 15th International Conference on Thalassaemia & Other Haemoglobinopathies & the 17th TIF Conference for Patients & Parents, that is held virtually on 19-21 November 2021.

Organised by the Thalassaemia International Federation (TIF), this conference promises to provide an outstanding opportunity for all involved stakeholders to bridge perspectives from various disciplines and to meet the challenges of improving the health and quality of life of those affected by haemoglobinopathies. This conference constitutes a unique forum for sharing knowledge and experiences and for building up new friendships, collaborations, partnerships and networks.

Following the global pandemic of COVID-19, this year’s conference requires a different approach, to allow international engagement and ensure participant safety. For the first time our International Conference will be web-based. To make sure that the conference will maintain its top-quality educational level despite its virtual nature, we aim to organize an exciting, ground-breaking and highly educational scientific program, covering a broad range of thematic topics.

Due to the virtual nature of the conference, for the first time the Scientific Sessions are joint with the Patients Programme and promise very interesting lectures and discussions that appeal to all scientists, patients and parents who deal with Thalassaemia and other Haemoglobin Disorders. We are very excited to take this initiative.

We very much look forward to welcoming and seeing you all at this event, aiming to build a brighter future together for patients with haemoglobinopathies all over the world!

Cordially,

Mr Panos EnglezosPresident,

Thalassaemia International Federation (TIF)

Dr Androulla EleftheriouExecutive Director,

Thalassaemia International Federation (TIF)

4

19-21 November 2021 Virtual

Committees

Conference Chairperson

Panos EnglezosPresident, Thalassaemia International Federation (TIF)

Organising Committee

Androulla EleftheriouExecutive Director, Thalassaemia International Federation (TIF)

Michael AngastiniotisMedical Advisor, Thalassaemia International Federation (TIF)

International Scientific Advisory Committee

Maria Domenica CappelliniProfessor of Haematology, Department of Medicine and Medical Specialties, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Foundation Maggiore Policlinico Hospital, Milan, Italy

Dimitrios FarmakisAssociate Professor, Cardiology, University of Cyprus Medical School, Nicosia, Cyprus

John KoskinasProfessor in Medicine - Hepatology, 2nd Academic Department of Medicine, Medical School of Athens, National and Kapodistrian University of Athens, Hippokration General Hospital, Athens, Greece

Marina CavazzanaProfessor of Haematology, Director of Dept. of Biotherapy at Necker-Enfants Malades Hospital, Paris Descartes-Sorbonne Paris Cité University, Director of INSERM/ Assistance Publique - Hospitals of Paris GHU Ouest Biotherapy Clinical Investigation Centre, Paris, France

Antonio PigaProfessor of Paediatrics & Dean of the Medicine School, San Luigi University Hospital, Turin University, Turin, Italy

John PorterProfessor of Haematology, Head of the Joint Red Cell Unit for UCLH & Whittington Hospitals, Haematology Department, University College London, London, UK

Stefano RivellaProfessor of Pediatrics - Children’s Hospital of Philadelphia (CHoP), University of Pennsylvania, Kwame Ohene-Frempong Chair on Sickle Cell Anemia, Philadelphia, USA

Ashraf SolimanProfessor of Pediatrics and Endocrinology, Hamad General Hospital, Doha, Qatar



Ali TaherProfessor of Medicine, Hematology & Oncology Director – Naef K. Basile Cancer Institute Vice Chair – Research, Department of Internal Medicine Founding Director – Fellowship and Residents Research Program, Faculty of Medicine American University of Beirut Medical Center, Beirut, Lebanon

Vip ViprakasitResearch Director, Thalassaemia Center and Division of Haematology-Oncology Department of Paediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand

Evangelia YannakiAffiliate Professor, University of Washington, Seattle Director, Gene and Cell Therapy Center, Hematology Dpt-BMT Unit G.Papanicolaou Hospital, Thessaloniki, Greece

International Patients/Parents Advisory Committee

HH Sheikha Sheikha Bint Seif Al-NahyanTIF Board Member, Emirates Thalassemia Society, United Arab Emirates

Michelle Abi SaadTIF Board Member, Chronic Care Center, Lebanon

Saeed Al AwadhiTIF Board Member - Ass. Treasurer, Emirates Thalassemia Society, United Arab Emirates

Angelo Loris BrunettaTIF Board Member, Associazione LigureThalassemici, Italy

George ConstantinouTIF Board Member - Ass. Secretary, UK

Riyad ElbardTIF Board Member – Treasurer, Thalassaemia Foundation of Canada, Canada

Robert FicarraTIF Board Member, Cooley’s Anemia Foundation, USA

Fatemeh HashemiTIF Board Member, Charity Foundation for Special Diseases, Iran

Abdul Baset Mohd MerdasTIF Board Member, Emirates Thalassemia Society, United Arab Emirates

Eleni MichalakiTIF Board Member, Greek Thalassaemia Federation, Greece

Elena MylonaTIF Board Member - Secretary, Cyprus Thalassaemia Association, Cyprus

Anton SkafiTIF Board Member, Thalassemia Patients Friends Society (TPFS), Palestine

Shobha TuliTIF Board Member - Vice President, Thalassemics India, India

*Names are in alphabetical order based on surname

6


Miguel AbboudPaediatric Haematology/Oncology, Department of

Paediatrics and Adolescent Medicine, American University of Beirut Medical Centre, Beirut,

Lebanon

Israa Al TaglubeePhysician, Patient Advocate, Al-Karama teaching hospital, Iraq

Ministry of Health, Baghdad, Iraq

Michelle Abi SaadTIF Board Member,

Administrative Director, Chronic Care Centre, Beirut, Lebanon

Evangelos AlexiouConsultant Radiologist, CT - MRI Department, General Hospital of

Larissa, Larissa, Greece

Hanny Al-SamkariClassical Hematologist and

Clinical Investigator, Associate Director, Hereditary Hemorrhagic

Telangiectasia Center, Massachusetts General Hospital, Assistant Professor of Medicine,

Harvard Medical School, USA

Michael AngastiniotisMedical Advisor, Thalassaemia International Federation (TIF)

Faculty Members



Kenneth AttiePaediatrician, Senior Vice

President & Chief Medical Officer, IMARA Inc.

Wilma BarcelliniProfessor of Haematology,

Responsible of Pathophysiology of Anaemias Unit, Istituto di Ricovero e Cura a Carattere

Scientifico (IRCCS) Ca’ Granda Foundation Maggiore Policlinico

Hospital, Milan, Italy

Angeliki BalassopoulouChief Biologist, Laboratory of

Genetics, Center of Expertise of Rare Haematological Diseases,

Laikon Hospital, Athens, Greece

Loris Angelo BrunettaTIF Board Member, Associazione

Ligure Thalassemici, Italy

Prebo BarangoMedical Officer Non-

Communicable Diseases, Inter-country Support Team for East

and Southern Africa, -WHO AFRO

Lily CannonTIF Operations Manager

8


Giles CampionHead of R&D and Chief Medical

Officer, Silence Therapeutics

George ConstantinouTIF Board Member -

Ass. Secretary, UK

Maria D. CappelliniProfessor of Haematology,

Department of Medicine and Medical Specialties, Istituto

di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda

Foundation Maggiore Policlinico Hospital, Milan, Italy

Halima DahmaniPhysician, Patient Advocate,

Blood Transfusion Center, Annaba, Algeria

Soteroula ChristouMedical Officer 1st Class, Medical

Services, Archbishop Makarios III Hospital, Thalassaemia Clinic,

Nicosia, Cyprus

Lucia de FranceschiAss. Prof., Internal Medicine,

University of Verona, Verona, Italy



Geoffrey DusheikoEmeritus Professor of Medicine,

Royal Free Hospital and University College London

School of Medicine; Consultant Hepatologist at King’s College

Hospital, London, UK

Maaike EijgenraamPyruvate Kinase Deficiency Patient, Joure, Netherlands

Stefan EberSpecial Praxis for Pediatric

Hematology and Childrens' Hospital, Technical University,

Munich, Germany

Riyad ElbardTIF Board Member – Treasurer, Thalassaemia Foundation of

Canada, Canada

Lakshman EdirisingheDirector, National Blood

Transfusion Service, WHO Collaborating Centre, Sri Lanka

Androulla EleftheriouExecutive Director, Thalassaemia

International Federation (TIF)

10


Perla EleftheriouConsultant Haematologist, Joint

Red Cell Unit, Haematology Department, Clinical Lead for

Red Cell Haematology,Honorary Clinical Senior Lecturer,

University College London, London, UK

Robert FicarraBoard Member – Thalassaemia International Federation (TIF), Vice-President of International

Affairs, Cooley’s Anemia Foundation, USA

Panos EnglezosPresident, Thalassaemia

International Federation (TIF)

Mehregan Hadipour DeshalPharmacist, Iranian Thalassaemia

Society, Iran

Dimitris FarmakisAssociate Professor, University

of Cyprus Medical School, Consultant Cardiologist, Nicosia,

Cyprus

Maria HadjidemetriouExecutive Board Member,

Cooley's Anemia Foundation, USA



Suradej HongengProfessor of Paediatrics, Chief

of Haematology Oncology, Department of Paediatrics,

Faculty of Medicine Ramathibodi Hospital, Mahidol University,

Bangkok Thailand

Fazalena JohariSecretary, Federation of Malaysia Thalassaemia Societies, Malaysia

Adlette InatiProfessor of Clinical Medicine, Lebanese American University School of Medicine, Consultant

Hematologist, Chronic Care Center, Head of Pediatric

Hematology Oncology and Director of Comprehensive Sickle

Cell Program at Nini Hospital – Tripoli, Beirut, Lebanon

Antonis KattamisProfessor of Paediatric

Haematology - Oncology, Head of Division of Paediatric Haematology-Oncology, First

Department of Paediatrics, National and Kapodistrian

University of Athens, 'Aghia Sophia' Children's Hospital,

Athens, Greece

Zsuzsanna JakabDeputy Director-General, World

Health Organisation, Geneva, Switzerland

John KoskinasProfessor in Medicine -

Hepatology, 2nd Academic Department of Medicine, Medical School of Athens, National and Kapodistrian

University of Athens, Hippokration General Hospital,

Athens, Greece

12


Dimitris KountourasInternist - Hepatologist, "MITERA"

Hospital, Athens, Greece

Carsten LedererScientist, Molecular Genetics Thalassaemia Department,

Cyprus Institute of Neurology & Genetics, Ass. Professor, Cyprus School of Molecular Medicine,

Nicosia, Cyprus

Carl LanderInternational Collaboration

Director, Thrive with PK Deficiency Organization, UK

Franco LocatelliProfessor of Paediatrics, Sapienza,

University of Rome, Head of the Department of Paediatric Haematology and Oncology,

IRCCS Bambino Gesù Children's Hospital, Rome, Italy

Stella KyriakidesEuropean Commissioner, Health & Food Safety, Brussels, Belgium

Dimitris LoukopoulosEmeritus Professor of

Haematology, First Department of Medicine at the University of

Athens, Athens, Greece



Alberto MartinezSenior Director, Clinical Science,

Silence Therapeutics

Ayesha MehmoodPresident of Fight Against

Thalassaemia (FAiTH) Foundation, Karachi - Pakistan

Yuyun MaryuningsihMedical Doctor, Specialisation in Transfusion Medicine, Team

Lead, Blood and other Products of Human Origin (BTT), World

Health Organization

Miltos MiltiadousPresident of the Board, Cyprus

Thalassaemia Association, Nicosia, Cyprus

Vikram MathewsProfessor of Haematology, Head of Department of Haematology,

Christian Medical & Hospital Vellore, India

Irene MottaAss. Prof. Internal Medicine,

Department of Clinical Sciences and Community Health, General Medicine & Rare Diseases Unit,

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Foundation Maggiore

Policlinico Hospital, Milan, Italy

14


Khaled MusallamGroup Chief Research Officer,

VPS Healthcare, Abu Dhabi, UAE, President, International Network

of Haematology, London, UK

John OldEmeritus Reader in Haematology, University of Oxford, Oxford, UK

Elena MylonaTIF Board Member - Secretary,

Cyprus Thalassaemia Association, Cyprus

Costas PapageorgiouPresident P.E.P.P.A.M.A, Former

President Thalassaemia International Federation (TIF),

Athens, Greece

Isaac OdameProfessor, Alexandra Yeo Chair

in Haematology, Director of the Division of Haematology,

Departments of Medicine and Paediatrics, University of

Toronto Haematology Section Head, Division of Haematology/

Oncology Medical Director, The Global Sickle Cell Disease

Network, Centre for Global Child Health The Hospital for Sick

Children

Alessia PepeCardiologist and Radiologist,

Magnetic Resonance Imaging Unit, Fondazione G. Monasterio C.N.R.- Regione Toscana 1, Pisa,

Italy



Mary PetrouConsultant Clinical Molecular

Geneticist & Head of Haemoglobinopathies Genetics

Services, University College London Hospitals (UCLH), Honorary Senior Lecturer,

Institute of Women's Health, University College London,

London, UK

Viresh PiplaniThalassemia Patients Advocacy

Group, New Delhi, India

Eshghi PeymanProfessor of Pediatric

Hematology & Oncology, Managing Director of Iranian

Blood Transfusion Organization (IBTO), WHO Collaborating

Centre, Shahid Beheshti University of Medical Sciences,

Tehran, Iran

Constantina PolitisProfessor Emeritus of

Medicine, National and Kapodistrian University of

Athens, Haematologist, Scientific Advisor to the

Hellenic National Public Health Organisation(EODY) for the

Coordinating Haemovigilance Centre and Surveillance of

Transfusion(SKAEM), Athens, Greece

Antonio PigaProfessor of Paediatrics & Dean

of the Medicine School, San Luigi University Hospital, Turin

University, Turin, Italy

John PorterProfessor of Haematology, Head

of the Joint Red Cell Unit for UCLH & Whittington Hospitals,

Haematology Department, University College London,

London - UK

16


Simona RasoDepartment of Haematology and Rare Diseases, V. Cervello, Azienda Ospedaliera Ospedali

Riuniti Villa Sofia-Cervello, Palermo, Italy

Tamara SchryverPresident & Co-founder, Thrive

with Pyruvate Kinase Deficiency, USA

Stefano RivellaProfessor of Paediatrics, Children’s Hospital of Philadelphia (CHoP),

University of Pennsylvania, Kwame Ohene-Frempong

Chair on Sickle Cell Anemia, Philadelphia, Pennsylvania, USA

Nicos SkordisProfessor of Paediatrics, School of

Medicine, University of Nicosia

Farrukh ShahConsultant Haematologist,

Department of Haematology, Whittington Hospital, Medical

Director - Transfusion, NHS Blood & Transplant, Chair UK Forum on

Haemoglobin Disorders, Chair National Haemoglobinopathy

Registry, London, UK

Pietro SodaniScientific Consultant, Haemato-

Oncology Paediatric Section, Bone Marrow Transplant Unit,

Charitè Hospital Berlin Germany & Azienda Ospedali Riuniti Marche Nord, Pesaro, Italy



Merula SteagallSocial Entrepreneur and Health

Advocacy Leader, President Brazilian Association for

Thalassaemia (ABRASTA) and Association for Lymphoma &

Leukemia (ABRALE), Sao Paolo, Brazil

Paul TelferProfessor of Clinical

Haematology, Queen Mary University of London, Honorary Consultant Haematologist, Barts

Health NHS Trust, London, UK

Ali TaherProfessor of Medicine,

Hematology & Oncology, Director – Naef K. Basile Cancer Institute Vice Chair – Research,

Department of Internal Medicine Founding Director – Fellowship

and Residents Research Program, Faculty of Medicine American

University of Beirut Medical Center Beirut, Lebanon

Evangelos TerposProfessor of Haematology,

University of Athens, School of Medicine and Director of

the Stem Cell Transplantation Unit, Department of Clinical

Therapeutics, Alexandra General Hospital, Athens, Greece

Anubha Taneja MukherjeeJoint Secretary, Thalassemics

India, Member-Secretary, Thalassemia Patients Advocacy

Group, New Delhi, India

Joanne Traeger-SynodinosAssociate Professor of Genetics,

Department of Medical Genetics, National and Kapodistrian

University of Athens, Choremeio Research Laboratory, 'Agia Sofia'

Children's Hospital, Athens, Greece

18


Shobha TuliVice-President – Thalassaemia International Federation (TIF),

Secretary – Thalassemics India, India

Malcolm WalkerProfessor of Cardiology, Consultant Cardiologist,

University College Hospital, Clinical Director Hatter

Cardiovascular Institute, London, UK

Eduard Van BeersHaematologist,

Van Creveldkliniek, University Medical Center,

Utrecht, Netherlands

Alejandra WatsonPresident and Co-Founder,

The Pyruvate Kynase Deficiency Foundation, USA

Vip ViprakasitProfessor of Paediatrics & Director

of Thalassaemia Research, Thalassaemia Center and Division

of Haematology-Oncology, Department of Paediatrics, Faculty of Medicine, Siriraj

Hospital, Mahidol University, Bangkok, Thailand

Evangelia YannakiAffiliate Professor, University of Washington, Seattle Director, Gene and Cell Therapy Center, Haematology Dept-BMT Unit,

G.Papanicolaou Hospital, Thessaloniki, Greece



Anne YardumianTIF Educational Advisor,

Consultant Haematologist, North Middlesex Hospital, North

Middlesex University Hospital NHS Trust, London

Manal ZaatarEgyptian Thalassemia Association

(ETA), Member of TIF’s Patient Advocacy Group, Cairo, Egypt

20


Friday, 19 November 2021

EET time Main Hall

11.45 - 13.15 Session 1: Challenges & Unmet Needs: the Patient Perspective

Chairs: E. Mylona & S. Tuli

Are we near achieving optimal care in:

11.45 - 11.55 EuropeG. Constantinou

11.55 - 12.05 Middle EastI. Al Taglubee

12.05 - 12.15 South East AsiaV. Piplani

12.15 - 12.25 West PacificF. Johari

12.25 - 12.35 North AmericaR. Elbard

12.35 - 12.45 South AmericaM. Steagall

12.45 - 13.15 Q&A

13.15 - 13.30 Comfort Break

13.30 - 14.45 PRESIDENTIAL SYMPOSIUM Organised by Thalassaemia International FederationThe Global Unmet Needs of Patients with Haemoglobin Disorders

Chairs: P. Englezos, C. Papageorgiou, R. Ficarra

13.30 - 13.45 Chronic Diseases and SDG2030 GoalsP. Barango

13.45 - 14.00 Haemoglobin Disorders & MigrationM. Angastiniotis

14.00 - 14.15 Combating Unmet Needs: TIF’s contributionA. Eleftheriou

14.15 - 14.45 Panel Discussion


15.00 - 16.00 Opening CeremonyChair: L. Cannon

15.00 - 15.30 Opening RemarksZ. Jakab, Deputy Director-General, World Health OrganisationS. Kyriakides, European Commissioner, Health & Food Safety P. Englezos, President of Thalassaemia International Federation



15.30 - 16.00 TIF Award Ceremony


16.15 - 18.15 MODERATED KEYNOTE SESSION: Haemoglobin Disorders in 2030

Chairs: A. Piga & G. Constantinou

Panelists:M. Angastiniotis, MD. Cappellini, G. Constantinou, A. Eleftheriou, P. Englezos, D. Loukopoulos, A. Mehmood, J. Porter, A. Taher, A. Mukherjee, V. Viprakasit & M. Zaatar


18.30 - 20.00 Session 2: Living with Thalassaemia & Sickle Cell Disease

Chairs: G. Constantinou & H. Dahmani

18.30 - 18.45 Balancing education, employment, work, social life and healthM. Miltiadous

18.45 - 19.00 Building truthful relationships – How and when do I tell my partner that I have a haemoglobin disorder?A. Mukherjee

19.00 - 19.15 How do I tell my child I have thalassaemia and what it meansM. Hadjidemetriou

19.15 - 19.30 My thoughts, challenges and fears during COVID-19 pandemicL. Brunetta

19.30 - 20.00 Q&A

22


Saturday, 20 November 2021

EET time Main Hall

09.30 - 11.00 Session 3: Clinical Management of Thalassaemia - TIF Guidelines Recommendations for TDT & NTDT

Chairs: G. Dusheiko & D. Farmakis

09.30 - 09.45 Blood TransfusionF. Shah

09.45 - 10.00 Iron Monitoring and ChelationJ. Porter

10.00 - 10.15 Cardiac ComplicationsM. Walker

10.15 - 10.30 Liver DiseaseJ. Koskinas

10.30 - 10.45 Bone DiseaseE. Terpos

10.45 - 11.00 Q&A


11.15 - 12.45 Session 3 continued: Clinical Management of Thalassaemia – TIF Guidelines Recommendations for TDT & NTDT

Chairs: A. Yardumian & J. Porter

11.15 - 11.30 Renal DiseaseA. Taher

11.30 - 11.45 ThrombosisMD. Cappellini

11.45 - 12.00 PainP. Eleftheriou

12.00 - 12.15 Fertility & PregnancyN. Skordis

12.15 - 12.30 Addressing Emergency ManagementJ. Porter

12.30 - 12.45 Q&A


13.00 - 14.30 Abstract Presentations Session

Chair: M. Angastiniotis

13.00 - 13.10 Development and Quality Improvement Assessment of a Thalassemia Care ProgramS. Fritch Lilla



13.10 - 13.20 The Iran thalassemia prevention program: A maxim for other countriesM. Hadipour

13.20 - 13.30 Diagnostic performance of a deep learning system for liver iron concentration measurement and comparison to a widely available T2* methodM. House

13.30 - 13.40 The International Hemoglobinopathy Research Network (INHERENT): An International Initiative to Study the Role of Genetic Modifiers in HemoglobinopathiesK. Kuo

13.40 - 13.50 Genome Editing for Beta-Haemoglobinopathies Without Double-Strand DNA CleavageN. Papaioannou

13.50 - 14.00 The ClinGen Hemoglobinopathy Variant Curation Expert PanelC. Stephanou

14.00 - 14.10 Efficacy and toxicity of Luspatercept in the treatment of Transfusion Dependent ThalassemiaK. Toutoudaki

14.10 - 14.30 Q&A


14.45 - 16.15 Session 4: Prevention of Haemoglobin Disorders

Chairs: D. Loukopoulos & J. Old

14.45 - 15.00 Where are we today in the prevention of haemoglobin disordersM. Angastiniotis

15.00 - 15.15 Challenges in implementing National Prevention Programmes in the control of Haemoglobin Disorders: the global perspectiveM. Petrou

15.15 - 15.30 Potential sources of error in population screening for haemoglobin disorders A. Balassopoulou

15.30 - 15.45 New Laboratory approaches for Haemoglobin DisordersJ. Traeger-Synodinos

15.45 - 16.15 Q&A


16.30 - 18.25 Session 5: Innovation in Haemoglobin Disorders

Chairs: A. Taher & A. Kattamis

16.30 - 16.50 Authorised and ongoing clinical trials of medical products & devices for the treatment of ThalassaemiaS. Rivella

24


16.50 - 17.10 Authorised and ongoing clinical trials of medical products & devices for the treatment of Sickle Cell DiseaseP. Eleftheriou

17.10 - 17.30 Exploring the Potential of Gene Silencing in Thalassemia G. Campion & A. Martinez

17.30 - 17.50 IMR-687, an Investigational PDE9 Inhibitor for SCD and Beta-thalassemia K. Attie

17.50 - 18.15 Q&A


18.30 - 20.00 Satellite Symposium - Supported by Pyruvate Kinase Activation: Update on Clinical Trial Results across Hemolytic Anemias

Moderator/Chair: MD. Cappellini

18.30 - 18.40 Welcome and IntroductionsMD. Cappellini

18.40 - 19.05 Mitapivat in pyruvate kinase deficiency – results of pivotal clinical trialsW. Barcellini

19.05 - 19.25 Activation of wild-type pyruvate kinase in thalassemia – clinical trial resultsMD. Cappellini

19.25 - 19.40 Activation of wild-type pyruvate kinase in sickle cell disease – preliminary findingsE. van Beers

19.40 - 20.00 Q&A and concluding remarks



Sunday, 21 November 2021

EET time Main Hall

11.00 - 13.00 Session 6: Sickle Cell Disease

Chairs: A. Inati & I. Odame

11.00 - 11.15 Sickle Cell Disease in ChildhoodI. Odame

11.15 - 11.30 Sickle Cell Disease in AdulthoodL. de Franceschi

11.30 - 11.45 Effective management of Pain CrisesA. Inati

11.45 - 12.00 Emeregency CareP. Telfer

12.00 - 12.25 Essential elements of Multidisciplinary Care in Sickle Cell DiseaseM. Abboud

12.25 - 12.50 Comparative Analysis of Guidelines/Standards for the Clinical Management of Sickle Cell DiseaseK. Musallam

12:50 - 13.10 Interactive Q&A Session moderated by European SCD Federation


13.15 - 14.30 Ice-Breaking Session 7: TIF’s Case Presentation Platform

Chairs: F. Shah & P. Eleftheriou

13.15 - 13.30 Complex Autoimmune Haemolytic Anaemia in Transfusion Dependent ThalassaemiaF. Shah

13.30 - 13.45 Liver Complications in ThalassaemiaD. Kountouras

13.45 - 14.00 Finding solutions for chelator side effectsS. Christou

14.00 -14.15 Pregnancy in SCDS. Raso

14.15 - 14.30 Q&A


14.45 - 16.30 Session 8: Curative Approaches in Haemoglobin Disorders

Chairs: E. Yannaki & P. Sodani

14.45 - 15.05 Haploidentical Transplantation with Post-Cyclophsphamide in ThalassemiaS. Hongeng

26


15.05 - 15.25 Haematopoietic Stem Cell Transplantation: Experience from IndiaV. Mathews

15.25 - 15.45 Haploidentical transplantation in Thalassaemia & SCDP. Sodani

15.45 - 16.05 Advances in Gene Therapy by Gene Addition (TDT and SCD)F. Locatelli

16.05 - 16.25 Advances in Gene Therapy by Genome Editing (TDT and SCD)C. Lederer

16.25 - 16.30 Q&A


16.45 - 18.00 Session 9: Emerging Concepts and Challenges in Haemoglobin Disorders

Chairs: MD. Cappellini & A. Kattamis

16.45 - 17.00 Ageing in Haemoglobin DisordersI. Motta

17.00 - 17.15 Solid & Haematological MalignanciesA. Taher

17.15 - 17.30 Haemoglobin Disorders and COVID-19: A global snapshotMD. Cappellini

17.30 - 17.45 The importance of genotype/phenotype in clinical decisions: Does one size fit all?S. Christou

17.45 - 18.00 Q&A


18.15 - 18.30 Closing Remarks

Chair: P. Englezos & A. Eleftheriou

A new era is dawning for the cure and care of Haemoglobin Disorders. Will 50 years of knowledge and experience find political commitment for the implementation of radical policy changes?



Monday, 22 November 2021

EET time Main Hall

10.00 - 11.00 TIF Consensus Meeting 1: Accurate Iron Load Measurement (ILM)

Chair: A. Pepe

10.00 - 10.30 How accuracy and reliability are ensured in Iron Load Monitoring?

10.00 - 10.10 An overview of the best practices in clinical care to date:The Cardiologist’s perspective M. Walker

10.10 - 10.20 An overview of the best practices in clinical care to date:The Hepatologist’s perspective D. Kountouras

10.20 - 10.30 An overview of the best practices in clinical care to date:The MRI Specialist’s perspective E. Alexiou

10.30 - 10.35 Why is this a concern for TIF?M. Angastiniotis & A. Eleftheriou

10.35 - 10.55 Setting standards for accurate ILM. Is it possible?Panel Discussion

10.55 - 11.00 ConclusionsA. Eleftheriou & A. Pepe


11.15 - 12.15 TIF Consensus Meeting 2: Chelation Drugs: Their Safety and Quality

Chair: M. Hadipour & A. Eleftheriou

11.15 - 11.25 How is safety and quality of chelation drugs defined?J. Porter

11.25 - 11.35 Is the continuous supply of drugs an element of safety?M. Angastiniotis & M. Abi Saad

11.35 - 11.50 What is the place and value of Generic formulations in a Healthcare System and what are the elements of safety involved in their use? The Thalassaemia perspective M. Hadipour

11.50 - 12.00 A CALL FOR ACTION NEEDED. What does this involve?A. Eleftheriou & M. Angastiniotis

12.00 - 12.10 Interactive Discussion

12.10 - 12.15 ConclusionsA. Eleftheriou & M. Hadipour


28


12.30 - 13.30 TIF Consensus Meeting 3: Blood Safety & Adequacy

Chair: C. Politis

12.30 - 12.45 Global overview of Blood Safety and Availability - The work of the WHO over the years on a recurring global issueY. Maryuningsih

12.45 - 13.05 The work of the WHO Blood Reference Centres in Asia and the Middle East RegionsThe Sri Lankan ExperienceL. EdirisingheThe Iranian ExperienceP. Eshghi

13.05 - 13.20 The global patients’ perspective, TIF’s 2021 Global Review and the need for an International Alliance of Patients for Blood Safety and AdequacyA. Eleftheriou

13.20 - 13.30 Interactive Discussion

14.00 - 17.00 TIF Constitutional Assembly by invitation only


17.15 - 18.00 TIF Board Meeting by invitation only

18.00 - 20.00 Building Best Practices in the Inherited Haemolytic Anaemias Community: Pyruvate Kinase Deficiency – Then, Now and the FutureChairpersons: H. Al Samkari & C. Lander

18.00 – 18.30 60 years since the first documented case: An overview of scientific advancements H. Al Samkari

18.30 – 18.50 Living with Pyruvate Kinase Deficiency through the years – the Patient PerspectiveM. Eijgenraam

18.50 – 19.10 Pyruvate Kinase Deficiency: A Rare Genetic Disease That Affects Red Blood Cells S. Eber

19.10 – 19.30 Building an organized patient community. A glimpse into current initiatives. PKD Foundation, A. WatsonThrive with PKD, T. Schryver

19.30 – 20.00 Q&A

When a disease is rare, listening shouldn’t be.

Listening to, collaborating with, and working for people with rare diseases drives everything we do as a company. Your struggles and successes fuel our passion, help us grow, and inspire us to do more.

That’s why we are committed to letting your voice shape how we develop resources and programs needed to face the challenges of living with a rare disease.

Chiesi Global Rare Diseases is proud to support the 15th International Conference on Thalassaemia & Haemoglobinopathies & the 17th TIF International Conference for Patients and Parents.

©CHIESI FARMACEUTICI S.p.A., 2021. All rights reserved. | PP-G-0489 V1.0

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About the Thalassaemia International Federation (TIF):

Thalassaemia International Federation, a non-governmental, patient driven umbrella organisation, established in 1986, supports today, the rights of patients for access to quality health, social and other care through its work with over 200 national thalassaemia associations in 62 countries across the world. Founded by a small group of doctors and patients/parents who represented National Patient Associations, mainly from Cyprus, Greece, Italy, UK and USA i.e. Countries where thalassaemia had been recognized early as a genetic, hereditary disorder with huge medical, public health, social and economic repercussions if left unaddressed in terms of both effective prevention and manage-ment. Thus, these were the countries where strong research activity was initiated and the first control programmes were implemented in the early 1980s, with measurable success. The rationale of these founding members lay on the establishment of an international umbrella organisation to build on the accumulated experience and the knowledge gained, aiming to support the efforts of other coun-tries since by the mid-1980s the worldwide prevalence of the disease had been well verified.

Our Mission: The prioritisation of thalassaemia on national health agendas and the development and implementation of effective disease – specific control (prevention and clinical management) programmes within national healthcare systems based on universal coverage

Our Vision: To support the provision of equal access of every patient with thalassaemia to high qual-ity health, social and other care in a truly patient-centred healthcare setting

Our Values: Transparency, reliability, ethos, accountability, independence and patient-centredness

Our Work: √ Education √ Advocacy √ Collaborations/ Networking √ Research √ Raising Awareness

Our Partners: > World Health Organisation: in official relations since 1996 > United Nations: in special consultative status with the United Nations Economic and Social Council (ECOSOC) since 2017

> European Union:• participatory status at the Council of Europe since 2019• official partners of the European Commission in the field of Health since 2018

Our Motto: Unity & Knowledge constitute our Strength!

More information at www.thalassaemia.org.cy

19-21 November 2021 - tifeducation.org

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Transcript of 19-21 November 2021 - tifeducation.org