Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study

Angchaisuksiri P.von Mackensen S.Apte S.Benson G.Eichler H.Findley A.Matsushita T.Mazini Tavares C.M.Puggaard Ravn M.Sathar J.Villarreal Martinez L.Young G.Mahidol University2025-03-232025-03-232025-02-01Research and Practice in Thrombosis and Haemostasis Vol.9 No.2 (2025)https://repository.li.mahidol.ac.th/handle/123456789/106760Background: Patient-reported outcomes (PROs) can provide useful insights into patient perception of concizumab, an anti–tissue factor pathway inhibitor monoclonal antibody intended for once-daily, subcutaneous prophylaxis for hemophilia A (HA) or hemophilia B (HB), with and without inhibitors. Objectives: To evaluate PROs from the phase 3 explorer8 study (NCT04082429). Methods: Male patients aged ≥12 years with HA/HB without inhibitors were enrolled and randomized 1:2 to no prophylaxis/on-demand treatment (arm 1) or concizumab prophylaxis (arm 2) or allocated to concizumab prophylaxis (arms 3 and 4). PRO questionnaires included the 36-item short-form health survey version 2, Haemophilia Quality of Life Questionnaire for Adults, Hemophilia Treatment Experience Measure, and Haemophilia Patient Preference Questionnaire. Results: Estimated treatment difference for change in 36-item short-form health survey version 2 “bodily pain” and “physical functioning” from baseline to week 24 between patients in arms 1 and 2 was 9.5 points (95% CI, 2.4 to 16.7) and 0.3 points (95% CI, −5.1 to 5.6), respectively. Estimated treatment difference at week 24 between patients in arms 1 and 2 was −18.0 points (95% CI, −26.4 to −9.5) for Haemophilia Quality of Life Questionnaire for Adults “total score” and −16.8 points (95% CI, −32.2 to −1.4) for “physical health.” Hemophilia Treatment Experience Measure and Haemophilia Patient Preference Questionnaire results favored concizumab prophylaxis over no prophylaxis or previous treatment. Conclusion: PRO data from the phase 3 explorer8 study provided additional support for concizumab prophylaxis compared with no prophylaxis as a treatment option for patients with HA/HB.MedicineConcizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 studyArticleSCOPUS10.1016/j.rpth.2025.1027052-s2.0-8600079670224750379