The potential impact of gene therapy on health-related quality of life (HRQoL) domains in haemophilia

Abstract

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.

Keywords

• haemophilia
• gene therapy
• patient-reported outcomes
• health-related quality of life