Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study
1
Issued Date
2025-02-01
Resource Type
eISSN
24750379
Scopus ID
2-s2.0-86000796702
Journal Title
Research and Practice in Thrombosis and Haemostasis
Volume
9
Issue
2
Rights Holder(s)
SCOPUS
Bibliographic Citation
Research and Practice in Thrombosis and Haemostasis Vol.9 No.2 (2025)
Suggested Citation
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. Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. Research and Practice in Thrombosis and Haemostasis Vol.9 No.2 (2025). doi:10.1016/j.rpth.2025.102705 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/106760
Title
Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study
Author's Affiliation
Ramathibodi Hospital
Belfast Health and Social Care Trust
Nagoya University Hospital
Universitätsklinikum des Saarlandes Medizinische Fakultät der Universität des Saarlandes
Keck School of Medicine of USC
Novo Nordisk A/S
Universitätsklinikum Hamburg-Eppendorf
Sahyadri Hospitals
Dr José Eleuterio González Monterrey University Hospital
Ampang Hospital
Belfast Health and Social Care Trust
Nagoya University Hospital
Universitätsklinikum des Saarlandes Medizinische Fakultät der Universität des Saarlandes
Keck School of Medicine of USC
Novo Nordisk A/S
Universitätsklinikum Hamburg-Eppendorf
Sahyadri Hospitals
Dr José Eleuterio González Monterrey University Hospital
Ampang Hospital
Corresponding Author(s)
Other Contributor(s)
Abstract
Background: 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.