Highlights From the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders 2025
Developing Evidence-Based Guidelines for AAV Gene Therapy in Hemophilia
Presenting Author: Glenn Pierce, MD, PhD
Pierce GF,1 Gouider E,2 Kaeser GE,3 Frantsve-Hawley J,4 Clairmont S,1 Coffin D1
- World Federation of Hemophilia, Montreal, Canada
- World Federation of Hemophilia, Tunis, Tunisia
- Gwen Kaeser Consulting, Bend, Oregon, USA
- EBQ Consulting Corp, Chicago, USA
Overview
Figure 1: Overview of Living Guidelines start with a Living Systematic Review (LSR)
The WFH has introduced a new chapter on the management of hemophilia with AAV gene therapy within the 3rd edition of the WFH Guidelines for the Management of Hemophilia, originally published in August 2020. The WFH assembled a diverse panel comprising 71% health care professionals and 29% patients from 18 countries. A systematic literature search identified 71 relevant studies, which informed the guideline development process. Panelists were divided into 10 topic-based subgroups focusing on key aspects of gene therapy management, including site preparedness, eligibility, screening, counseling, infusion day, efficacy, durability, safety, post-gene therapy monitoring, and lifestyle choices. The panel employed a modified Delphi process, reaching a consensus on 85 recommendations after three rounds of review. As part of its ongoing efforts to keep clinical guidelines up to date, the WFH plans to next revise the Treatment and Prophylaxis chapters from the 2020 guidelines.
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