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DOI: 10.1055/s-0041-1730283
Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease
Funding The study was funded by Octapharma AG. Medical writing assistance was provided by nspm ltd, Meggen, Switzerland, and funded by Octapharma AG.![](https://www.thieme-connect.de/media/10.1055-s-00033990/202103/lookinside/thumbnails/10-1055-s-0041-1730283_210004-1.jpg)
Abstract
Background The efficacy and safety of wilate (human von Willebrand factor/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD.
Objectives The objectives of this observational, prospective, phase 4 study were to evaluate the safety, tolerability, and effectiveness of wilate in on-demand treatment of bleeding episodes (BEs), long-term prophylaxis, and surgical prophylaxis among patients with any type of VWD.
Methods Patients were enrolled at 31 study centers in 11 countries and followed for up to 2 years. Safety endpoints included adverse drug reactions (ADRs) and drug tolerability. Effectiveness was assessed using annualized bleeding rates (ABRs) during prophylaxis and predefined criteria for the treatment of BEs and surgical prophylaxis.
Results A total of 111 patients (76 [68%] female) including 41 (37%) children were treated with wilate. Twenty-five patients received prophylaxis, 29 on-demand treatment, and 62 surgical prophylaxis. Tolerability was rated by patients as “excellent” for 96.2% of 6,497 infusions. No unexpected ADRs or thrombotic events were reported. Median ABR during prophylaxis was 1.9. Effectiveness was assessed as “excellent” or “good” by patients and investigators for 100% of BEs treated on-demand, 98% (patient rating) and 99% (investigator rating) of breakthrough BEs, and 99% of surgical procedures (investigator rating).
Conclusion wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.
Keywords
factor VIII - observational study - prophylaxis - von Willebrand disease - von Willebrand factorAuthor Contributions
All authors made substantial contributions to the acquisition and interpretation of the data for the work. All authors critically revised the manuscript for important intellectual content, approved the final version submitted, and agreed to be accountable for all aspects of the work.
Publication History
Received: 30 October 2020
Accepted: 17 March 2021
Article published online:
04 July 2021
© 2021. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)
Georg Thieme Verlag KG
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