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CC BY 4.0 · TH Open 2021; 05(03): e264-e272
DOI: 10.1055/s-0041-1730283
Original Article

Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease

Michelle Sholzberg
1   Departments of Medicine and Laboratory Medicine & Pathobiology, St. Michael's Hospital, Li Ka Shing Knowledge Institute, University of Toronto, Toronto, Canada
2   Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, Ontario, Canada
,
Kate Khair
3   Research in Children's Health, Illness and Disability (ORCHID), NIHR Biomedical Research Centre, Great Ormond Street Hospital for Children, London, United Kingdom
,
Hassan Yaish
4   University of Utah School of Medicine, Salt Lake City, Utah, United States
,
George Rodgers
4   University of Utah School of Medicine, Salt Lake City, Utah, United States
,
Maria Sol Cruz
5   Fundación de la Hemofilia de Salta, Salta, Argentina
,
Cesar Montaño Mejía
6   Hemolife and Universidad Tecnológica de Pereira, Pereira, Colombia
,
Zuzana Čermáková
7   Blood Center, University Hospital Ostrava, Ostrava-Poruba, Czech Republic
,
Davide Matino
8   McMaster University Medical Center, Hamilton, Ontario, Canada
,
Jerome Teitel
2   Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, Ontario, Canada
,
Alpha Barrie
3   Research in Children's Health, Illness and Disability (ORCHID), NIHR Biomedical Research Centre, Great Ormond Street Hospital for Children, London, United Kingdom
,
Sylvia Werner
9   Octapharma USA, Inc., Paramus, New Jersey, United States
,
Mario von Depka Prondzinski
10   Werlhof-Institut GmbH, Hannover, Germany
› Author Affiliations Funding The study was funded by Octapharma AG. Medical writing assistance was provided by nspm ltd, Meggen, Switzerland, and funded by Octapharma AG.
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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 factor

Author 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.


Supplementary Material



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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