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

Angchaisuksiri P 1 , von Mackensen S 2 , Apte S 3 , Benson G 4 , Eichler H 5 , Findley A 6 Show all authors , Matsushita T 7 , Mazini Tavares CM 8 , Puggaard Ravn M 9 , Sathar J 10 , Villarreal Martinez L 11 , Young G 12

Affiliations 

  • 1 Division of Haematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
  • 2 Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany
  • 3 Department of Haematology, Sahyadri Specialty Hospitals, Pune, India
  • 4 Department of Haematology, Belfast Health and Social Care Trust, Belfast, Northern Ireland, United Kingdom
  • 5 Institute of Clinical Haemostaseology and Transfusion Medicine, Saarland University and University Hospital, Homburg, Germany
  • 6 Medical & Science Patient Focused Drug Development, Novo Nordisk A/S, Søborg, Denmark
  • 7 Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan
  • 8 Medical & Science Rare Bleeding Disorders, Novo Nordisk A/S, Søborg, Denmark
  • 9 Biostatistics RD & AT 1, Novo Nordisk A/S, Søborg, Denmark
  • 10 Department of Haematology, Ampang Hospital, Kuala Lumpur, Malaysia
  • 11 Department of Haematology, Dr. José Eleuterio González Monterrey University Hospital, Monterrey, Nuevo León, México
  • 12 Hemostasis and Thrombosis Centre, Children's Hospital of Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California, USA
Res Pract Thromb Haemost, 2025 Feb;9(2):102705.
PMID: 40166710 DOI: 10.1016/j.rpth.2025.102705

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.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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