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Jour 1 | 4 novembre : SAFETY, SUPPLY AND ACCESS TO TREATMENT PRODUCTS

9h00 - 9h10

WELCOME REMARKS
Glenn Pierce, Vice-President Medical, World Federation of Hemophilia, San Diego, United States
Radoslaw Kaczmarek, Coagulation, Products Safety, Supply and Access Committee, World Federation of Hemophilia, Indianapolis, United States

9h10 - 10h30

PLASMA AND ACCESS
Moderator
Mark Skinner, President and CEO, Institute for Policy Advancement Ltd, Washington, United States

10h30 - 11h50

SAFETY
Moderator
Brian O'Mahony, CEO, Irish Haemophilia Society, Dublin, Ireland

11h50 - 12h10

Pause

12h10 - 13h30

TREATMENT QUESTIONS
Moderator
Johnny Mahlangu, Professor, University of the Witwatersrand and NHLS, Johannesburg, South Africa

13h30 - 14h45

VON WILLEBRAND DISEASE
Moderator
Baiba Ziemele, President, Latvia Hemophilia Society, Riga, Latvia

 

Jour 2 | 5 novembre : RESEARCH AND HEMOPHILIA TREATMENT

9h00 - 10h20

REAL WORLD DATA
Moderator
Alfonso Iorio, Professor, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, Canada

10h20 - 11h40

GENE THERAPY
Moderator
David Lillicrap, Professor, Queen's University, Kingston, Canada

11h40 - 12h00

Pause

12h00 - 13h15

ABSTRACT PRESENTATIONS
Moderator
Glenn Pierce, Vice-President, Medical, World Federation of Hemophilia, La Jolla, United States

13h15 - 13h25

CLOSING REMARKS
Glenn Pierce, Vice-President Medical, World Federation of Hemophilia, San Diego, United States
Radoslaw Kaczmarek, Coagulation, Products Safety, Supply and Access Committee, World Federation of Hemophilia, Indianapolis, United States

LES HORAIRES INDIQUÉS CORRESPONDENT À L'HEURE DE MONTRÉAL (EASTERN TIME)
LE PROGRAMME PEUT FAIRE L'OBJET DE CHANGEMENT

 

WELCOME REMARKS / PLASMA AND ACCESS

The first session of the 12th WFH Global Forum will welcome participants and address issues related to plasma and access to treatment. Speakers will discuss access to treatment and the impact of COVID-19, plasma safety and residual pathogen risk, and the DDAVP intranasal shortage.

Welcome Remarks

Glenn Pierce, Vice-President Medical, World Federation of Hemophilia, San Diego, United States
Radoslaw Kaczmarek, Coagulation, Products Safety, Supply and Access Committee, World Federation of Hemophilia, Indianapolis, United States


Moderator

Mark Skinner, President and CEO, Institute for Policy Advancement Ltd, Washington, United States

Geopolitical access to treatment and COVID-19 impact
Brian O'Mahony, CEO, Irish Haemophilia Society, Dublin, Ireland

Plasma safety and residual pathogen risk with plasma-derived clotting factors
Svetoslav Slavov, Young Investigator FAPESP, Hemocentro Ribeirão Preto, São Paulo, Brazil

Current safety issues and observations
Thomas Kreil, Head of Global Pathogen Safety, Takeda, Vienna, Austria

DDAVP Intranasal shortage and alternatives
Steve Pipe, Professor of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States
Dan Hart, Consultant Haematologist, The Royal London Hospital Haemophilia Centre, United Kingdom
Magdy El-Ekiaby, Senior Consultant Hematology, Shabrawishi Hospital Hemophilia Centre, Cairo, Egypt

SAFETY

This session will address a range of safety issues such as defining and reporting issues, post hepatitis C monitoring and long-term follow-up & thrombosis risk with non-replacement therapies. The session will be moderated by Brian O’Mahony.

Moderator

Brian O'Mahony, CEO, Irish Haemophilia Society, Dublin, Ireland


What constitutes a safety issue: what should be reported? when should reports be issued? by whom and to whom?
Len Valentino, CEO, National Hemophilia Foundation, New York, United States

Non-replacement therapies and risk of thrombosis
Mike Makris, Professor, University of Sheffield, United Kingdom

Post hepatitis C monitoring and long term follow-up
Bruce Luxon, Chair, Department of Medicine - Georgetown University, Washington, D.C, United States

ATHN-7: long term follow-up of non-factor replacement therapies
Michael Recht, Chief Science Officer, American Thrombosis and Hemostasis Network, Rochester, United States

TREATMENT QUESTIONS

Addressing some of the latest treatment questions, experts in this session will discuss the extravascular pool of factor 9, how it is distributed and clinical implications for EHLs, starting emicizumab in previously untreated patients, as well as the subcutaneous delivery of factor.

Moderator

Johnny Mahlangu, Professor, University of the Witwatersrand and NHLS, Johannesburg, South Africa


Extravascular distribution of factor 9
Darrel Stafford, Professor of Biology, University of North Carolina, Chapel Hill, United States

Extravascular pool of factor 9: clinical implications for EHLs
Robert Sidonio, Pediatric Hematologist/Oncologist, Aflac Cancer and Blood Disorders, Atlanta, United States

Starting emicizumab immediately: How should 50 EDs of PUPs be planned in the era of emicizumab?
Gili Kenet, Director, Israel National Hemophilia Center and Thrombosis Institute, Ramat Gan, Israel

Subcutaneous delivery of factor
Johnny Mahlangu Professor, University of the Witwatersrand and NHLS, Johannesburg, South Africa

VON WILLEBRAND DISEASE

The new von Willebrand Disease (VWD) treatment guidelines will be discussed, from limitations to implementation into practice. In addition, novel treatment options and advances in diagnostic testing will be presented.

Moderator

Baiba Ziemele, President, Latvia Hemophilia Society, Riga, Latvia


Novel treatment modalities for VWD
Cécile Denis, Director Unit 1176, INSERM/Université Paris-Sud/Université Paris-Saclay, Paris, France

VWD prophylaxis - learning from Haemophilia and not having undue delay
James O'Donnell, Professor of Vascular Biology, Royal College of Surgeons, Ireland

Limitations of the VWD guidelines
Paula James, Professor Hematologist, Department of Medicine, Queen's University, Kingston, Canada

New initiatives in VWD diagnostic tests
Angela Weyand, Assistant Professor, Department of Pediatrics, University of Michigan, Ann Arbor, United States

REAL WORLD DATA

In this session on Real World Data, experts will discuss how data-driven decisions help improve patient care for people with bleeding disorders. Different WFH initiatives will be presented.

Moderator

Alfonso Iorio, Professor, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, Canada


WFH Treatment Guidelines: Setting the Standards of Care
Donna Coffin, Director, Research & Education, World Federation of Hemophilia, Canada

Humanitarian Aid initiatives based upon the WFH Treatment Guidelines
Jay Martin, Development & Training Manager - Humanitarian Aid, World Federation of Hemophilia, Canada

Measuring outcomes with WBDR and GTR
Donna Coffin, Director, Research & Education, World Federation of Hemophilia, Canada

Annual Global Survey: capturing new products
Jeff Stonebraker, Associate Professor, North Carolina State University, Raleigh, United States

How patient reported level data can be used to compare therapies before and after switching
Martin Scott, Consultant Haematologist, Manchester University Hospitals NHS Foundation Trust, Manchester, United Kingdom

GENE THERAPY

As a novel therapeutic, gene therapy raises new safety issues, implications and challenges. These will all be discussed in this session moderated by Dr. David Lillicrap.

Moderator

David Lillicrap, Professor, Queen's University, Kingston, Canada


Importance of integration as a potential safety risk or unknown
Paul Batty, Post Doctoral Fellow, Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada

Duration of steroid therapy and its complications in gene therapy
Margareth Ozelo, Professor, Hemocentro Unicamp, University of Campinas, Brazil

Development of hepatocellular carcinoma
Heiner Wedemeyer , Professor and Chairman of the Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Germany

Review on measurement of FVIII and IX post gene therapy
Annette Bowyer, Senior Biomedical Scientist, Sheffield Teaching Hospitals NHS FT, United Kingdom

ABSTRACT PRESENTATIONS

Moderator

Glenn Pierce, Vice-President, Medical, World Federation of Hemophilia, La Jolla, United States


The protocol for your gene therapy clinical trial in LMIC
Ulrike Reiss, Associate Member, St. Jude Children's Research Hospital, Memphis, United States

Long-term Safety and Efficacy of the Anti-TFPI Monoclonal Antibody Marstacimab in Patientswith Severe Haemophilia A or B: Results from a Phase 2 Long-term Treatment Study
Johnny Mahlangu Professor, University of the Witwatersrand and NHLS, Johannesburg, South Africa

Clinical Development of Efanesoctocog Alfa (BIVV001), a New Class of Factor VIII (FVIII) Replacement Therapy
Roshni Kulkarni, Professor & Director, Centers for Bleeding and Clotting Disorders, Michigan State University, East Lansing, Michigan

Update on Fitusiran
Baisong Mei, Senior Global Project Head, Rare Diseases and Rare Blood Disorder Clinical Development, Sanofi, Cambridge, United States

CLOSING REMARKS

Réseau de la FMH

FMH
La FMH joue un rôle de premier plan dans le financement et l'amélioration des soins aux personnes souffrant de troubles héréditaires de la coagulation.
FMH USA
La FMH USA fait progresser la cause de la FMH aux États-Unis.
Plateforme d’apprentissage en ligne
Un accès facile à toutes les ressources de la FMH sur les troubles de la coagulation.
Le monde de l’hémophilie
Découvrez les actualités de la FMH et de la prise en charge des troubles de la coagulation dans le monde.

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Nous contacter

  • Secrétariat du Forum mondial
    Fédération mondiale de l’hémophilie
    1425, boul. René-Lévesque Ouest, Bureau 1200 
    Montréal, Québec H3G 1T7 Canada
    Tel.: +1 (514) 875-7944
    Fax: +1 (514) 875-8916
    Courriel: [email protected]