Real-World Unmet Treatment Needs for Patients With Haemophilia: Results From the Global Adelphi Disease Specific...

Prophylactic factor replacement and non-factor therapy has improved bleed prevention and clinical outcomes for people with haemophilia (PwH). The remaining burdens faced by PwH in real-world settings, despite availability of newer therapies, are not well documented.

We aimed to describe the haemophilia patient population, assess clinical and humanistic burden of disease, and identify unmest needs associated with current treatments.

Data were drawn from the Adelphi Real World Haemophilia Disease Specific Programme, a cross-sectional survey of haemophilia-treating physicians, their patients with haemophilia A (HA) or B (HB), and caregivers. Data were collected in multiple countries from July 2023 to October 2024. Physicians reported patient demographics, disease characteristics, treatment details, bleeding episodes and joint health status. Patients and caregivers reported health-related quality of life (HRQoL). Analyses were descriptive.

Overall, 283 physicians reported on 2227 patients, with 67% of PwH aged 18-49 years. Prophylactic compliance was high: 94% of patients were either fairly or fully compliant (taking 50%-‍80% or >80% of prescribed dose, respectively). However, physicians were not completely satisfied with prophylaxis for 56% of patients with HA and 59% with HB, citing concerns about administration methods, dosing schedules, and efficacy. Joint problems were experienced by 49% of patients; 57% reported ≥1 bleed annually despite prophylaxis. HRQoL impairment was reported by patients on all types of prophylaxis.

Despite current treatment, many PwH continue to experience limitations to their daily activities, productivity, and physical independence. A patient-centred approach is needed to ensure clinical effectiveness can be balanced with lifestyle priorities.