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

Key Data Points

WFH GTR Implementation

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.

WFH GTR Objectives

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.

RELATED CONTENT

Interactive Webinars
Image

Please enable the javascript to submit this form

Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

Essential SSL