Gene therapy for people with Haemophilia B: a proposed care delivery model in Italy.

BACKGROUND: Gene therapy is designed to provide people with haemophilia B with a steady and elevated factor IX activity, thereby strengthening protection and relieving the burden of replacement therapy frequent infusions. The European Medicines Agency has approved a gene therapy for the severe and moderately severe forms of haemophilia B that uses the factor IX Padua variant (etranacogene dezaparvovec). The aim is to provide a document dedicated to haemophilia B gene therapy and give a comprehensive overview of the topic.

METHODS: An Italian group of experts in haemophilia has carried out a narrative review of the literature and has discussed during a virtual meeting several key aspects of the delivery of this treatment in Italy. The discussion covered the organisational model, the role of the multidisciplinary team, the laboratory surveillance and the patient journey, from the follow-up to the identification of safety issues and outcome measures.

RESULTS: This article highlights the need to follow the Hub and Spoke organisational model and sheds light on the role of each professional figure within the multidisciplinary teams to favour patient engagement, management, and retention. Moreover, this article stresses the need to perform laboratory tests for patient screening and follow-up and proposes a checklist to help patient identification. Finally, the needs of Italian haemophilia centres have been considered to ensure an efficient implementation of the care delivery model.

CONCLUSION: It is crucial to ensure that centres are appropriately organized, equipped and trained to adequately select patients, deliver the gene therapy and perform follow-up.