The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025

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

As gene therapy becomes more widely available for hemophilia, healthcare providers need clear guidance on how to use it safely and effectively. The World Federation of Hemophilia (WFH) developed the first international guidelines for adeno-associated virus (AAV) gene therapy to help standardize care and support informed decision-making for patients, caregivers, and healthcare teams.

The guidelines provide evidence-based recommendations for hemophilia treatment centers, clinicians, and people with hemophilia, with a strong focus on patient safety, long-term monitoring, and consistent care. Because gene therapy is a rapidly evolving field, the guidelines will be updated as new research becomes available.

AAV gene therapy; bleeding disorders; haemophilia; management guidelines; monitoring; outcomes; shared decision‐making