PB0212 - Collaboration between the World Federation of Hemophilia and National Registries for the Long-Term Follow-up of People with Hemophilia Treated with Gene Therapy

Sunday, July 10, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

MN

Mayss Naccache

Gene Therapy Program Manager
World Federation of Hemophilia
Montreal, Quebec, Canada
Barbara A. Konkle, MD

Professor of Medicine
University of Washington
Seattle, Washington, United States

Presenting Author(s)

Donna Coffin, MSc Epid

Director, Research & Education
World Federation of Hemophilia
Montreal, Quebec, Canada

Co-Author(s)

CC

Cary Clark

Director of Programs and Education
International Society on Thrombosis and Haemostasis
Carrboro, North Carolina, United States
LG

Lindsey A. George

Assistant Professor of Pediatrics
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
AI

Alfonso Iorio

Professor
Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON
Hamilton, Ontario, Canada
Wolfgang Miesbach, MD

Professor and Head of Coagulation Disorders and Comprehensive Care Centre
University Hospital Frankfurt, Frankfurt, Germany
Frankfurt, Hessen, Germany
DN

Declan Noone

President
European Haemophilia Consortium
Brussels, Brussels Hoofdstedelijk Gewest, Belgium
Flora Peyvandi, MD, PhD

Professor of Internal Medicine; Director of the Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Milan, Lombardia, Italy
Steven W. Pipe, MD

Professor of Pediatrics and Pathology
University of Michigan, Ann Arbor, MI, USA
Ann Arbor, Michigan, United States
Michael Recht, MD, PhD, MBA

Chief Science Officer
American Thrombosis and Hemostasis Network
Rochester, New York, United States
MS

Mark W. Skinner

President
Institute for Policy Advancement Ltd.
Washington, District of Columbia, United States
LV

Leonard A. Valentino

Physician
Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois, USA
New York, New York, United States
Johnny Mahlangu, MD, MMed

Professor in Haematology
University of the Witwatersrand and NHLS
Johannesburg, South Africa
GP

Glenn F. Pierce

VP Medical
World Federation of Hemophilia
Montreal, Quebec, Canada
Frank W.G. Leebeek, MD, PhD

Professor and Head of Department of Hematology
Erasmus MC, University Medical Center Rotterdam, The Netherlands
Rotterdam, Zuid-Holland, Netherlands

Background: Registries, with international collaboration between countries, are an effective way to pool enough data to enable robust evaluation of rare safety events and durability of gene therapy. Linking registries is an extensive process involving many steps and collaboration among multiple partners.

Aims: To maximize the utility of current data collection systems, the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) is collaborating with national registries to promote international data integration.

Methods: The technical ease of linking registries relies on meticulous preparatory work between registries, to minimize data integration barriers. Barriers include poor interoperability due to the complexity and heterogeneity of data fields, lack of standard terms, data descriptions, and measurement methods.

A credible database partner, with technical capability and strong security systems has been selected and is central to a successful registry and data integration. De-identified data will be transferred from partner registries to the GTR on a regular basis, without access to any identifying information other than a unique patient identifier.

Results: The GTR has begun a collaboration with the American Thrombosis and Hemostasis Network (ATHN) (USA), HemoNed (Netherlands), and the Canadian Bleeding Disorders Registry (CBDR) (Canada). Common data definitions and careful comparison of data field measurements, such as ensuring common ontology terms, units of measurement and date formats, as much as possible, was conducted to facilitate the technical mapping of data fields between the GTR and partner registries. Discussions on potential integrations with other national registries are ongoing.

The GTR will be available for data entry and linkage by mid-2022.

Conclusion(s): The GTR is collaborating with ATHN, HemoNed, and the CBDR to ensure that rare adverse events, in a small patient population over a large geographical area, will be detected. The GTR and partner registries will also help establish long-term follow-up as the standard of care for gene therapy.