Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?

Authors: Sidonio, RF, JR; Corrales-Medina, FF; Johnsen, JM; Sholzberg, M; Malcolmson, C; Connell, NT

Affiliations: Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, 2220 North Druid Hills Road NE, Atlanta, GA 30329, USA. Division of Pediatric Hematology-Oncology, Department of Pediatrics, University of Miami, Miller School of Medicine, Miami, FL, USA. University of Miami-Hemophilia and Thrombosis Treatment Center, Miami, FL, USA. Department of Medicine, University of Washington, Seattle, WA, USA. Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA. Department of Medicine, St. Michael’s Hospital, Li Ka Shing Knowledge Institute, University of Toronto, Toronto, ON, Canada. Department of Laboratory Medicine and Pathobiology, St. Michael’s Hospital, Li Ka Shing Knowledge Institute, University of Toronto, Toronto, ON, Canada. Division of Haematology/Oncology, The Hospital for Sick Children, University of Toronto, Department of Paediatrics, Toronto, ON, Canada. Division of Hematology, Department of Medicine, Brigham and Women’s Hospital, Boston, MA, USA. Harvard Medical School, Boston, MA, USA.

Publication: Therapeutic Advances in Hematology; 2026; 17. 20406207261431617

ABSTRACT: A deficiency and/or dysfunction of von Willebrand factor (VWF) or factor VIII (FVIII) results in the bleeding disorders of von Willebrand disease (VWD) and hemophilia A (HA), respectively. Whereas HA impacts coagulation, VWD primarily impairs hemostasis through defective platelet adhesion and aggregation. In addition, because VWF protects FVIII from proteolytic degradation, a deficiency in VWF can also reduce FVIII levels and affect coagulation. While regular prophylaxis to restore FVIII activity is the standard of care for severe HA, its use to correct the dual VWF/FVIII defect in severe VWD is less well established. Current treatment guidelines suggest the use of long-term prophylaxis rather than no prophylaxis in persons with VWD with a history of severe and frequent bleeds. In this narrative review, we discuss the barriers to the broader adoption of prophylaxis in persons with severe VWD and results of recent clinical studies that provide further evidence to support its use. A growing body of evidence suggests that prophylaxis should be established as the standard care for individuals with severe VWD and recurrent bleeding.