PB0231 - Treatment satisfaction and joint health outcomes within a real-world haemophilia B population: the Adelphi Disease Specific Programme™ survey

Background: While haemophilia care continues to improve, people with haemophilia B (PwHB) may still experience disease and treatment burden.

Aims: To describe physician-reported treatment satisfaction and joint health outcomes in a real-world population of PwHB.

Methods: The Adelphi Real World haemophilia Disease Specific Programme™ is a retrospective, cross-sectional survey gathering data on treatment satisfaction and joint health via physician-completed patient record forms. Data were obtained from France, Germany, India, Italy, Japan, Spain, the UK and USA. Physician inclusion criteria were: (1) haematology or haematology-oncology specialist; (2) treats ≥5 patients with haemophilia A/B per month. PwHB inclusion criteria were: (1) males with moderate-to-severe HB; (2) receiving extended half-life (EHL) or standard half-life (SHL) therapy prophylactically.

Results: In this interim analysis, 85 physicians provided data on 232 PwHB receiving prophylaxis with EHL (n=199 [86%]) or SHL therapy (n=33 [14%]). Demographics were similar between the two treatment groups (Table 1), although a greater proportion of people with severe HB received EHL therapy. Physicians reported that 84% of PwHB receiving EHL and 73% receiving SHL therapy were fully compliant (received >80% of prescribed dose). Physicians were not completely satisfied with the current treatment for 57% of patients receiving EHL and 70% receiving SHL therapy, for reasons including treatment schedules and lack of effectiveness for specific bleed types. Physicians reported 42% (n=83/199) of PwHB receiving EHL and 36% (n=12/33) receiving SHL therapy had joint problems due to haemophilia. In the overall PwHB group, physicians reported that 28% (n=64) had problems in ≥1 target joint and 36% (n=83) had joint pain (Table 2).

Conclusion(s): Although physicians reported good adherence to therapy among PwHB, most were not completely satisfied with treatment outcomes, and reported joint problems for more than one-third of PwHB. With this evaluation revealing scope for improvement, future analysis will provide detail regarding treatment satisfaction from the patients’ perspective.