Successful use of emicizumab prophylaxis in a patient with von Willebrand disease type 3: A case report

Von Willebrand disease (VWD) type 3 is the most severe form of VWD and is often inadequately controlled with plasma-derived von Willebrand factor (VWF)/FVIII or fresh frozen plasma. Emicizumab, a factor VIII– mimetic antibody approved for hemophilia A, enhances secondary hemostasis and has emerging off-label evidence in severe VWD. A 38-year-old woman with VWD type 3 and recurrent mucocutaneous bleeding despite regular factor therapy was started on emicizumab prophylaxis. Over 6 months, she remained bleed-free without additional factor support. Activated partial thromboplastin time normalized with increased measured FVIII activity; the apparent VWF antigen rise was interpreted cautiously. The treatment was well-tolerated and cost-effective. Emicizumab may offer an effective alternative prophylaxis in selected patients with severe VWD.