Hemophilia A Patient Satisfaction With Extended Half-life Altuviiio® Therapy

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.

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).

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.

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’, ‘reduction in bleeds and bleed-related complications’, 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.

Prophylactic efanesoctocog alfa was preferred to prior prophylactic regimens by most patients and improved patient satisfaction at 52 weeks.