Abstract
Introduction Haemophilia A is a rare bleeding disorder caused by defects in coagulation factor VIII (FVIII). Damoctocog alfa pegol (BAY 94-9027, Jivi, Bayer, Germany) is a site-specifically PEGylated, extended-half-life, recombinant FVIII, approved for use in previously treated patients (PTPs) aged ≥12 years with haemophilia A. However, a real-world evidence regarding routine clinical use of damoctocog alfa pegol is limited. Methods and analysis HEM-POWR is a multinational, multicentre, non-interventional, prospective, postmarketing cohort study evaluating the effectiveness and safety of real-world treatment with damoctocog alfa pegol. Estimated enrolment is ≥200 PTPs with haemophilia A, receiving damoctocog alfa pegol (on-demand, prophylaxis or intermittent prophylaxis (as per local label)), observed for 36 months. Primary outcomes are total bleeding events and annualised bleeding rate; secondary outcomes include long-term safety, joint health, pharmacokinetics, patient-reported outcomes (PROs) from validated questionnaires and perioperative haemostasis. Where applicable, reasons for switching to damoctocog alfa pegol, choice of treatment regimen and dose will also be captured. Exploratory and descriptive statistical analyses will be performed, and will be stratified by parameters including, but not limited to, prophylaxis regimen and haemophilia severity. Patients can record bleeds and consumption in electronic (e) Diaries, ePROs, and can access non-promotional study information (videos explaining study procedures) via an online patient portal. Optionally, patients can enrol in the LIFE-ACTIVE substudy designed to investigate the relationship between activity (measured by the ActiGraph CP Insight watch) and effectiveness parameters collected from HEM-POWR. Ethics and dissemination Study approval was obtained by local independent ethics committees and authorities in participating study centres across Europe, the Americas and Asia. Informed consent from patients or their legal representative is a requirement for participation. The study results will be submitted for publication in a peer-reviewed scientific journal and presented at scientific conferences. Trial registration numbers NCT03932201, EUPAS26416. Protocol version and date V.1.2, 27 September 2019.
| Original language | English (US) |
|---|---|
| Article number | e044997 |
| Journal | BMJ open |
| Volume | 11 |
| Issue number | 9 |
| DOIs | |
| State | Published - Sep 2 2021 |
Bibliographical note
Funding Information:1Department of Hematology, Bayer AG, Basel, Switzerland 2Hematology Department, Hospital Universitario La Paz, Madrid, Spain 3Center for Bleeding Disorders and Coagulation, Department of Oncology, University Hospital Careggi, Firenze, Italy 4Center for Bleeding and Clotting Disorders, Tulane University School of Medicine, New Orleans, Louisiana, USA 5Department of Blood Transfusion Service, Nagoya University Hospital, Nagoya, Japan 6Faculty of Medical Sciences, University Medical Centre Groningen, Groningen, Netherlands 7Institute of Experimental Haematology and Transfusion Medicine, University Hospital Bonn, Bonn, Germany 8Department of Oncology and Heamatology, Bayer AG, Berlin, Germany 9Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center, Minneapolis, Minnesota, USA Acknowledgements Critical review and statistical support were provided by Inga Bayh, an employee of Bayer AG. Medical writing support was provided by Rebekka Harding-Smith and Frank Biegun of Darwin Healthcare Communications (Oxford and London, UK), funded by Bayer.
Funding Information:
This work was supported by Bayer.
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Keywords
- bleeding disorders and coagulopathies
- clinical trials
- protocols and guidelines
- public health