On the 100th anniversary of von Willebrand disease’s discovery, experts gathered at EAHAD’s 2026 Congress to reflect on progress - and persistent mysteries.
Dr. James O’Donnell, National Coagulation Centre, Dublin, opened the Arosenius Lecture by tracing VWD’s evolution since 1926. Once a clinical curiosity in a 5-year-old girl, it’s now known to affect ~1% of the global population - characterized by mucocutaneous bleeding, heavy periods, and surgical complications.
VWF, synthesized in endothelial cells, forms multimers critical for platelet adhesion and Factor VIII stabilization. Four distinct VWF pools exist in the body - plasma, Weibel-Palade bodies, platelet α-granules, and vessel wall matrix - each playing unique hemostatic roles.
Despite 15,000+ PubMed publications, 2021 ASH/ISTH/NHF/WFH guidelines still rely heavily on low-certainty evidence. Nine of eleven diagnostic recommendations are “conditional.” Eight management guidelines are all “suggestions.”
Key gaps remain: Why do some with “low VWF” (30-50 IU/dL) bleed severely while others don’t? LoVIC and Zimmerman Program data show strong bleeding correlation in adult women - but not consistently in children. Overlap with “bleeding disorders of unknown cause” hints at undiscovered genetic modifiers.
Emerging science reveals VWF’s role beyond clotting: angiogenesis, wound healing, immune modulation. For women, consequences are severe - 40% miss work or school, 8% require hysterectomy.
New therapies in development: rondoraptivon pegol (BT200), KB-V13A12, HMB-002 - targeting specific pathways for precision care.
One hundred years later, biological discovery is accelerating. The next challenge: turning insight into individualized outcomes.