Describing the clinical outcomes and treatment landscape for male haemophilia A and B patients in a real-world setting across Europe and the United States

Haemophilia A and haemophilia B are rare bleeding disorders characterized by prolonged bleeding episodes, bruising, and spontaneous bleeds. As the treatment landscape evolves, real-world data are essential to assess disease management practices. This study aimed to describe clinical outcomes for haemophilia A and haemophilia B patients in a real-world setting. Data were drawn from the Adelphi Haemophilia Disease Specific Programme (DSP), a cross-sectional survey of physicians and their patients conducted in France, Germany, Italy, Spain, the United Kingdom, and the United States between February 2020 and May 2021. Physicians provided data on patient demographics, clinical characteristics, bleed history, and treatment patterns. Analyses were descriptive. Overall, 75 physicians reported data for 739 patients with haemophilia A and 131 patients with haemophilia B, with a mean [standard deviation (SD)] age of 27 (14.9) and 25.5 (15.8) years. At data collection, the most common treatment received by haemophilia A patients was emicizumab (40%), while for haemophilia B, this was extended half-life factor (55%). Since switching to their current prophylactic treatment, 48% of haemophilia A patients and 54% of haemophilia B patients had experienced one or more episodes of breakthrough bleeding. Of these patients, the most common bleed type experienced in the 12 months prior to data collection was joint bleeds (53% of haemophilia A patients and 57% of haemophilia B patients). These findings highlight that despite prophylactic treatment, breakthrough bleeding, particularly within the joints, remains common, emphasizing the need for more effective therapeutic strategies.