Von Willebrand disease.

Long-term prophylaxis is not as well known in Von Willebrand disease (VWD) as in hemophilia but attempts to evaluate prophylaxis scientifically in VWD have started. A few cohort studies have been reported. In an international effort the Von Willebrand disease prophylaxis network (VWD PN) has been formed to investigate the role of prophylaxis in clinically severe VWD (e.g., patients with type 3 VWD) that is nonresponsive to other treatments. Findings from the VWD PN studies will hopefully provide more robust evidence for which patients might best benefit from prophylaxis and for appropriate dosing regimens for prophylaxis in patients with VWD.