Advances in Von Willebrand Disease

Authors: Lassila, R; Atiq, F; Casari, C; Lavin, M

Affiliations: Research Program Unit in Systems Oncology, Helsinki University Hospital, University of Helsinki, Helsinki, Finland. Department of Haematology, Erasmus MC, Erasmus University Medical Center, Rotterdam, the Netherlands. Irish Centre for Vascular Biology, School of Pharmacy and Biomolecular Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland. Universite Paris-Saclay, INSERM, Hémostase Inflammation Thrombose HITh U1176, Le Kremlin-Bicêtre, France. National Coagulation Centre, St. James’s Hospital, Dublin, Ireland.

Publication: Haemophilia: the official journal of the World Federation of Hemophilia; 2025

ABSTRACT: In recent decades the advances in care and therapeutic options for people with haemophilia have been extraordinary, transforming the lives of those with severe disease and access to treatment. In contrast, the treatments for those living with von Willebrand Disease (VWD) were largely developed over 20 years ago, with the first recombinant VWF product only recently achieving a licence for use in prophylaxis. Marking the 100th anniversary of the first reported case of a person with VWD, PwVWD and their healthcare providers need to continue to demand and work toward improved access to both diagnostics and better treatment internationally for PwVWD. Herein we reflect on the evolution of knowledge and care for PwVWD and review the current approaches to diagnosis. The limited current therapeutic options for prophylaxis and burden of infusions remain a hindrance to broad use in PwVWD who would benefit. There is, however, a changing landscape for PwVWD as, for the first time in VWD, multiple novel therapeutic approaches are either in or approaching clinical trial development. With these advances in mind, we look forward with hope toward rapid and sustained improvements in the next century for all PwVWD.