Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.

Background: Treatments for Hemophilia A include prophylaxis with factor VIII (FVIII) replacement therapy. With standard and extended half-life therapies, patients still experience bleeds, and administration 2-4 times a week is needed, which affects adherence. Efanesoctocog alfa is a once-weekly FVIII replacement therapy approved to treat and prevent bleeding in patients with hemophilia A for all age groups. Objectives: To analyze patient treatment preferences and satisfaction comparing once-weekly efanesoctocog alfa with pre-study prophylaxis in adults and adolescents with severe hemophilia A in the pivotal Phase 3 XTEND-1 study (NCT04161495). Methods: Exploratory post-hoc patient-reported outcomes were assessed by treatment preference at Week 52 (via a treatment preference survey) and patient satisfaction at baseline and up to Week 52 (via the 9-item Treatment Satisfaction Questionnaire for Medication [TSQM-9], covering: global satisfaction, effectiveness and convenience domains). Relationships between treatment satisfaction and preference versus bleeding and region were also explored. Results: Of 130 patients who completed the preference survey, 90.0% preferred efanesoctocog alfa versus their previous prophylaxis regimen. Similar trends were observed by region. The most common reasons were 'less frequent treatment', 'bleeds and bleed-related complications reduction', and 'feel better protected'. Of 115 patients who completed the TSQM-9 at Week 52, there was a statistically significant improvement from baseline in all three domains. By study end, an increase in the proportion of patients selecting the "best response" for all TSQM-9 items was observed. There were no strong correlations with treatment satisfaction improvements at Week 52 and improvement in quality of life or bleeding episodes. Conclusions: Prophylactic efanesoctocog alfa was preferred to prior prophylactic regimens by most patients and improved patient satisfaction at 52 weeks.