haemophilia, gene therapy, patient-reported outcomes, health-related quality of life, Diseases of the blood and blood-forming organs, RC633-647.5

Haemophilia is an inherited bleeding disorder characterised by spontaneous bleeding, often leading to impaired health-related quality of life (HRQoL). Commonly used treatments include episodic and prophylactic treatment regimens. Gene therapies could soon become available, potentially creating a paradigm shift in patient management.