Open Access
CC BY 4.0 · Thromb Haemost
DOI: 10.1055/a-2665-2313
Review Article

Von Willebrand Factor as a Therapeutic Target in Thrombotic Disorders

1   Division of Thrombosis and Hemostasis, Department of Internal Medicine, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands
,
Bart J.M. van Vlijmen
1   Division of Thrombosis and Hemostasis, Department of Internal Medicine, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands
,
Jeroen C.J. Eikenboom
1   Division of Thrombosis and Hemostasis, Department of Internal Medicine, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands
› Author Affiliations

Funding This work was financially supported by the Dutch Thrombosis Foundation (TSN grant #2018-01).


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Abstract

Von Willebrand factor (VWF) plays an important role in primary hemostasis. Dysregulated plasma VWF levels are implicated in various pathological conditions. Reduced or dysfunctional VWF is associated with bleeding, known as von Willebrand disease. Whereas elevated plasma VWF levels may give rise to an increased risk of developing arterial thrombotic events. In general, antithrombotic strategies in arterial thrombosis primarily focus on inhibiting platelet aggregation; however, treatment failure, antiplatelet drug resistance, and adverse bleeding tendencies underscore the necessity for the development of more efficacious and safer therapeutic modalities. Targeting VWF presents an interesting therapeutic approach as it operates independently of platelet activation pathways for platelet-rich thrombus formation. Over time, several VWF inhibitors have progressed to clinical application for thrombosis management, with ongoing research endeavors exploring novel compounds targeting VWF. This review provides a comprehensive overview of the evolution of VWF-targeting therapeutic agents, elucidating their current developmental stages, clinical indications, and evaluating their respective advantages and limitations.



Publication History

Received: 17 February 2025

Accepted: 24 July 2025

Article published online:
11 August 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/)

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