The World Federation of Hemophilia Gene Therapy Registry

The World Federation of Hemophilia Gene Therapy Registry
Highlights From the 62nd ASH Annual Meeting and Exposition

The World Federation of Hemophilia Gene Therapy Registry

Barbara A. Konkle, MD1,2, Donna Coffin, MSc3, Mayss Naccache1, Robert Clark4, Lindsey George, MD5, Alfonso Iorio, MD, PhD6, Wolfgang A. Miesbach, MD7, Brian O'Mahony8, Flora Peyvandi9,10, Steven W. Pipe, MD11, Adrian Quartel12, Eileen K Sawyer, PhD13, Mark W Skinner, JD14, Bartholomew J. Tortella, MD, MBA15, Crystal Watson, BS16, Leonard A. Valentino, MD17, Ian Winburn18, Johnny Mahlangu19, and Glenn F. Pierce, MD, PhD20

1World Federation of Hemophilia, Montreal, QC, Canada

2University of Washington, Washington Center for Bleeding Disorders, Seattle, WA

3World Federation of Hemophilia, Montréal, QC, Canada

4International Society on Thrombosis and Haemostasis, Inc, Carrboro

5The Children’s Hospital of Philadelphia, Philadelphia, PA

6McMaster University, Ontario, Canada

7Hemophilia Centre, University Hospital Frankfurt, Frankfurt, Germany

8Irish Haemophilia Society, Dublin, Ireland

9Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

10Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

11Pediatrics and Pathology, University of Michigan, Ann Arbor, MI

12BioMarin Pharmaceutical Inc, Novato

13uniQure Inc, Lexington, MA

14Institute for Policy Advancement Ltd, Washington, DC

15Spark Therapeutics, Philadelphia, PA

16American Thrombosis and Hemostasis Network, Decatur, GA

17National Hemophilia Foundation (NHF), New York

18Pfizer Inc, New York

19Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg North, South Africa

20World Federation of Hemophilia, La Jolla, CA

Key Data Points

PHASE I/II STUDY DESIGN

Following guidance of the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA), the WFH GTR SC has developed this core set of data to be collected on all patients with hemophilia, who receive gene therapy. Collection of data will be requested quarterly during the first year post-gene therapy infusion and annually thereafter, over the patient’s lifetime. Outreach to HTCs began in August 2020 with initiation visits beginning in October 2020. The registry will go live in January 2021.

RELATED CONTENT