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The hub-and-spoke model in France: Organization of care and patient pathways for gene therapy in hemophilia

Par : Contributeur(s) : Type de matériel : TexteTexteLangue : français Détails de publication : 2023. Sujet(s) : Ressources en ligne : Abrégé : For decades, patients with hemophilia were treated using factor VIII (FVIII) and FIX concentrates. In the last fifteen years, hemophilia treatments have been tremendously improved through the development of extended half-life factors and novel treatment options, such as the bispecific antibody, emicizumab. Several therapeutic molecules targeting coagulation inhibitors, as well as FVIII mimetics, are currently under development. Gene therapy is another innovative approach that will soon be available on the market. The European Association for Haemophilia and Allied Disorders (EAHAD) and the European Haemophilia Consortium (EHC) recently recommended a new health-care framework for the management of patients set to receive gene therapy. This model, known as the hub-and-spoke model, aims to ensure the safe administration and monitoring of gene therapy by establishing close collaboration between treatment centers, the “hubs,” and local hemophilia treatment centers, the “spokes,” which will provide routine patient follow-up. According to this model, any adverse events should be managed by both centers (hub and spoke) to provide the timeliest state-of-the-art care for patients receiving gene therapy. In addition, all adverse events and efficacy data should be collected on a lifelong basis using a centralized registry. In France, the French Reference Center for Hemophilia (CRH) and the French Hemophilia Association (AFH) have worked together to adapt the European hub-and-spoke model in line with existing infrastructures and organizational procedures for rare bleeding disorders in France, which are grouped together under the French network on inherited bleeding disorders (MHEMO). This review describes their proposal for patient pathways and the organization of care for gene therapy in France.
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For decades, patients with hemophilia were treated using factor VIII (FVIII) and FIX concentrates. In the last fifteen years, hemophilia treatments have been tremendously improved through the development of extended half-life factors and novel treatment options, such as the bispecific antibody, emicizumab. Several therapeutic molecules targeting coagulation inhibitors, as well as FVIII mimetics, are currently under development. Gene therapy is another innovative approach that will soon be available on the market. The European Association for Haemophilia and Allied Disorders (EAHAD) and the European Haemophilia Consortium (EHC) recently recommended a new health-care framework for the management of patients set to receive gene therapy. This model, known as the hub-and-spoke model, aims to ensure the safe administration and monitoring of gene therapy by establishing close collaboration between treatment centers, the “hubs,” and local hemophilia treatment centers, the “spokes,” which will provide routine patient follow-up. According to this model, any adverse events should be managed by both centers (hub and spoke) to provide the timeliest state-of-the-art care for patients receiving gene therapy. In addition, all adverse events and efficacy data should be collected on a lifelong basis using a centralized registry. In France, the French Reference Center for Hemophilia (CRH) and the French Hemophilia Association (AFH) have worked together to adapt the European hub-and-spoke model in line with existing infrastructures and organizational procedures for rare bleeding disorders in France, which are grouped together under the French network on inherited bleeding disorders (MHEMO). This review describes their proposal for patient pathways and the organization of care for gene therapy in France.

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