Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Highlights From the WFH 2022 World Congress

Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Presented by: Barbara A. Konkle, MD, University of Washington, Seattle, Washington, USA

Barbara A. Konkle¹, Donna Coffin², Mayss Naccache², Cary Clark³, Lindsey A. George⁴, Alfonso Iorio⁵, Wolfgang Miesbach⁶, Declan Noone⁷, Flora Peyvandi⁸, Steven Pipe⁹, Michael Recht¹⁰, Mark Skinner¹¹, Leonard Valentino¹², Johnny N. Mahlangu¹³, Glenn F. Pierce²

¹University of Washington, Seattle, United States
²World Federation of Hemophilia, Montreal, Canada
³International Society on Thrombosis and Haemostasis, Carrboro, United States
⁴The Children’s Hospital of Philadelphia, Philadelphia, United States
⁵Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada
⁶Medical Clinic 2/Institute of Transfusione Medicine University Hospital Frankfurt, Germany
⁷European Haemophilia Consortium, Belgium
⁸Università degli Studi di Milano, Italy
⁹Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States
¹⁰American Thrombosis and Hemostasis Network, Rochester, United States
¹¹Institutefor Policy Advancement Ltd., and Department of Health Research Methods, Evidence, and Impact, McMaster University, Washington, District of Columbia
¹²National Hemophilia Foundation, New York, United States
¹³University of the Witwatersrand, South Africa

The WFH gene therapy registry (GTR) is a prospective, observational, and longitudinal registry with an overall goal of collecting data for all patients who receive gene therapy for hemophilia, both in clinical trials and in a post-marketing phase if and when approved. These are the primary and secondary objectives of the registry.

In order to implement the GTR, WFH is reaching out to individual hemophilia treatment centers (HTCs), starting with those involved in hemophilia gene therapy clinical trials. National hemophilia registries will also be asked to contribute. WFH is also working with industry to develop a process for obtaining data from completed clinical trials.