PB0219 - The World Federation of Hemophilia Living Guidelines Model

Background: The 3rd edition of the World Federation of Hemophilia’s (WFH) Guidelines for the Management of Hemophilia, published in 2020, includes over 300 practical recommendations for healthcare professionals and people with hemophilia. To ensure the recommendations remain up to date and current, the WFH has moved to a Living Guidelines Model (LGM). LGM is defined as the continuous monitoring of relevant literature ensuring recommendations are re-evaluated when important new evidence is published, ensuring recommendations are kept up to date.

Aims: The aim of the WFH LGM is to provide timely updates to the recommendations in the 3rd edition of the WFH Guidelines by updating individual recommendations when new evidence becomes available.

Methods: The LGM is being implemented by a multi-disciplinary Steering Committee (SC), a Guidelines Oversight Committee and topic-specific Panels. All groups are composed of healthcare and allied healthcare professionals, people with hemophilia and caregivers, ensuring global representation. The standard PICO methodology of updating guidelines is being used. Individual recommendations will be updated on a prioritization and rotational basis, established by the SC. The first topic selected centered on Adeno-Associated Virus (AAV) Gene Therapy (GT) for hemophilia A and B.

Results: The panelists received Training in guideline methodology and recommendation writing. Four research questions address safety, efficacy, quality of life, and site preparedness. The literature search (PubMed, EMBASE, and Cochrane Library) identified 36 eligible studies addressing one or more of the research questions. Recommendations will be related to treatment with GT and to follow-up care and management of patients who have received GT.

Conclusion(s): Developing and updating WFH recommendations using the LGM allows for a systematic prioritization of topics and evidence synthesis, with the unit of update as individual recommendations rather than chapters. The recommendations on AAV GT for hemophilia will be published in mid-2024.