Modèle hub & spoke en France : organisation des soins et parcours des patients pour la thérapie génique de l'hémophilie.

During several decades, patients with haemophilia have been treated using FVIII and FIX concentrates by haematologists working in specialized comprehensive haemophilia care centres. In the last fifteen years haemophilia treatments have been tremendously improved and novel treatment options have been developed. Gene therapy is one of these innovative approaches and will soon be available on the market. The European Association for Haemophilia and Allied Disorders and the European Haemophilia Consortium recently described a new healthcare setting for the management of patients who will receive gene therapy. This hub-and-spoke model aims to ensure the safe administration and monitoring of gene therapy by expert comprehensive haemophilia care centres, in close collaboration with the proximity haemophilia treatment centres where the patient is followed up. Any adverse events should be managed by both the expert "hubs" in collaboration with the spoke centre to provide the timeliest state-of-the-art treatment and management options to patients receiving gene therapy. All adverse events and efficacy data should be reported lifelong to a centralised registry. French reference centre for haemophilia and the patient's organization AFH worked together to adapt the European Hub and spoke model to the existing infrastructures and organization for rare bleeding disorders in France. This review describes the haemophilia patient's journey for gene therapy in France. [ABSTRACT FROM AUTHOR]