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CC BY 4.0 · TH Open 2025; 09: a26069625
DOI: 10.1055/a-2606-9625
Case Report

von Willebrand Factor (VWF) Inhibitors in Two Brothers with von Willebrand Disease: A Case Report

Claudia Djambas Khayat
1   Hotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon
,
Anna Pavlova
2   Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany
,
Sylvia Werner
3   Octapharma USA, Paramus, New Jersey, United States
,
Sigurd Knaub
4   Octapharma AG, Lachen, Switzerland
,
Robert F. Sidonio Jr
5   Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, United States
› Author Affiliations
Funding Financial support for medical writing services and article processing charges was provided by Octapharma AG, Lachen, Switzerland.
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Abstract

The development of inhibitors to von Willebrand factor (VWF) is a rare but potentially serious complication of VWF replacement therapy in patients with von Willebrand disease (VWD). Patients who develop VWF inhibitors may become unresponsive and/or may develop severe anaphylactic reactions to VWF concentrates. Data on inhibitor development and management in VWD remain limited, and better understanding of inhibitor development is an important goal in VWD management. The WIL-31 study demonstrated the efficacy and safety of prophylaxis with wilate, a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate, in children and adults with VWD of all types. The annualized bleeding rate (ABR) was reduced by 84% with wilate prophylaxis compared with on-demand treatment, and prophylaxis was well tolerated. No inhibitors developed during the WIL-31 study. Here, we report two brothers with type 3 VWD who at the 6-month visit were found to have VWF inhibitors, which on further investigation were found to have already been present before the study. Despite the presence of inhibitors, neither patient showed any clinical symptoms, and prophylaxis with wilate led to a ≥85% reduction in ABR in both boys compared with on-demand treatment.

Keywords

von Willebrand disease - plasma-derived von Willebrand factor - prophylaxis - von Willebrand factor inhibitors

Authors' Contributions

The initial draft of the manuscript was prepared by C.D.K. and R.F.S. All the authors contributed to the writing of the manuscript, provided critical amendments, and approved the final article for publication.


Data Availability Statement

Individual patient data and related documents (including study protocol and clinical study report) will be available on request.




Publication History

Received: 12 December 2024

Accepted: 24 April 2025

Article published online:
06 June 2025

© 2025. 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
Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

Bibliographical Record
Claudia Djambas Khayat, Anna Pavlova, Sylvia Werner, Sigurd Knaub, Robert F. Sidonio. von Willebrand Factor (VWF) Inhibitors in Two Brothers with von Willebrand Disease: A Case Report. TH Open 2025; 09: a26069625.
DOI: 10.1055/a-2606-9625
 

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