The commercialization of hemophilia AAV gene therapies in recent years has created a steep learning curve for clinicians, patients, and other stakeholders in the bleeding disorders community. As a novel treatment unlike any that has come before, gene therapy has compelled a series of questions and considerations relevant to its efficacy, safety, patient eligibility, implementation, cost/reimbursement, and more.
National health organizations, government agencies, and hemophilia treatment centers (HTCs) have all recognized the need for guidance and clarity relevant to all aspects of gene therapy including (but not limited to) HTC preparedness, screening/eligibility amongst patient subgroups, shared decision making, pre-and-post infusion management, potential adverse events, additional health considerations, clinical education/training, and the role of patient registries.
The World Federation of Hemophilia (WFH) had the foresight to form a WFH AAV Gene Therapy Panel, which consisted of experts in the field who were also investigators on the AAV clinical trials, healthcare teams currently caring for patients on gene therapy, as well as people with hemophilia and their caregivers. The panel created comprehensive and holistic guidelines to offer physicians and other prescribers access to the latest evidence-based clinical guidance and to help patients make more informed decisions about gene therapy. The authors noted that other stakeholders may also find utility in the guidelines, including payors, policymakers, local governments, patient advocacy organizations, and drug manufacturers.
These guidelines were further reviewed and endorsed by the European Haemophilia Consortium, the Coalition of the Americas, the Council of Ministers of Health of Central America and the Dominican Republic, and NBDF’s Medical and Scientific Advisory Council (MASAC). The authors noted that these guidelines will not remain static but will be revised on an ongoing basis as new evidence becomes available.
Access the full WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025 to learn more, including background on the process undertaken to create and finalize these important guidelines.
Citation
Pierce GF, Ozelo MC, Mahlangu J, Dunn AL, Beijlevelt M, Bender R, Carcao M, Coffin D, Grazzi EF, Foster GR, Frantsve-Hawley J, Iorio A, Jackson S, Kaczmarek R, Kaeser GE, Konkle BA, Lewandowska M, Miesbach W, O'Mahony B, Pipe S, Reiss UM, Wilson AK, Gouider E; WFH AAV Gene Therapy Guidelines Expert Panel. The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025. Haemophilia. 2025 Oct 25. doi: 10.1111/hae.70113. Epub ahead of print. PMID: 41137692.
NBDF, through its Neil Frick Resource Center, provides periodic synopses of articles published in peer-reviewed journals, the purpose of which is to highlight papers that cover a wide range of topics and relate to a broad spectrum of the inherited blood disorders community. Prior summaries have focused on topics such as shared decision-making, gene therapy, health equity, and more. NBDF hopes you find this content to be informative and engaging.
Disclaimer: Any questions about the articles featured here should be directed to the specific publisher, journal and/or authors. This content is being shared for general information only. NBDF does not give medical advice nor engage in the practice of medicine. NBDF under no circumstances recommends a particular treatment for specific individuals and in all cases recommends that you consult your physician or HTC before pursuing any course of treatment.
