Defining the Role of Nurses in Gene Therapy for Haemophilia

Haemophilia is a rare bleeding disorder now undergoing a major shift in care as gene therapy (GT), delivered through viral‑vector in vivo gene transfer, has enabled adults with haemophilia A or B to stop prophylaxis with minimal bleeding. As this treatment becomes a standard option in several European countries, nurses play an essential role in education, multidisciplinary care coordination, bleed management, and long‑term follow‑up, requiring ongoing professional development and potentially new specialist roles.

To describe and explore the multifaceted responsibilities of nurses throughout the GT pathway, emphasising their critical role in delivering holistic, patient-centered care.

This manuscript draws on discussions among experienced European haemophilia nurses to examine their expanding responsibilities throughout the GT pathways.

Nurses play a central role throughout the GT pathway in haemophilia, guiding shared decision‑making, preparing patients for infusion, delivering safe peri‑infusion care, and coordinating extensive post‑infusion monitoring. Their responsibilities include eligibility assessment, patient education, vigilant observation during infusion, early detection of adverse events, and long‑term follow‑up of liver function, factor levels, and treatment durability. They also support psychosocial adjustment, provide lifestyle counselling, uphold ethical communication about uncertainties, and ensure continuity of care across hub‑and‑spoke networks. In addition, nurses contribute to registries, quality improvement, and research, generating real‑world evidence essential for evaluating long‑term safety and effectiveness of GT.

GT in haemophilia requires well‑trained and well‑informed nurses to ensure safe delivery, effective follow‑up, and high‑quality patient support throughout the entire treatment pathway.