Late Wilate Prophylaxis Initiation in Adult Twins with Type 3 von Willebrand Disease: Overcoming Historical Barriers and Improving Clinical Outcomes

Abstract Prophylaxis with a plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII) 1:1 concentrate (wilate) is effective in the treatment of severe type 3 von Willebrand disease (VWD). However, uptake of prophylaxis with factor concentrates remains limited due in part to the treatment burden and historical concerns over blood product safety. We describe clinical cases of two adult twin brothers with severe type 3 VWD and a history of hepatitis C acquired from childhood plasma-derived products, who initiated once-weekly pdVWF/FVIII 1:1 concentrate prophylaxis after years of on-demand therapy. Both patients completed 12 months of prophylaxis, which led to a marked improvement in bleeding control and daily functioning, and a decrease in treatment burden compared with on-demand treatment. Their experiences highlight the significant enhancement in quality of life achieved through the adoption of regular prophylaxis. Overcoming long-standing concerns about product safety allowed them to embrace preventive care and realize its benefits in managing their severe VWD.