The WFH guidelines for the management of haemophilia: AAV gene therapy, 2025

The WFH guidelines for the management of haemophilia: AAV gene Therapy, 2025 - World Federation of Hemophilia (WFH)

Summary: 
As AAV gene therapy becomes more widely used in clinical practice, evidence-based guidelines with careful interpretation of the evidence by experts are essential to ensure patient safety, standardize clinical practices, address long-term uncertainties, support informed and shared decision-making and build stakeholder trust. This is the first iteration of the WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy. These guidelines were developed to provide comprehensive, evidence-based recommendations to support standardized, safe and effective implementation of gene therapy for haemophilia. They were developed by the WFH AAV Gene Therapy Panel, which is composed of gene therapy experts who were involved in clinical trials and healthcare teams currently treating patients who have received gene therapy, as well as people with haemophilia and their caregivers. Because evidence is rapidly evolving in the treatment landscape for haemophilia, particularly AAV gene therapy, these guidelines have been developed under the WFH LGM, and will be updated as new evidence becomes available.

As treatment with AAV gene therapy for haemophilia becomes more common, multiple stakeholders must work together to ensure a seamless and standardized implementation that prioritizes patient safety. Therefore, these guidelines have recommendations for haemophilia treatment centres, the comprehensive care medical team and people with haemophilia. Other stakeholders who may find these guidelines relevant are payors, policymakers, local governments, patient advocacy organizations and drug manufacturers.

These guidelines were reviewed by external parties of interest and have been endorsed by the European Haemophilia Consortium (EHC), the Coalition of the Americas, the Council of Ministers of Health of Central America and the Dominican Republic (COMISCA), and the Medical and Scientific Advisory Council (MASAC) of the National Bleeding Disorders Foundation.


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