The role of prophylaxis in the management of von Willebrand disease: today and tomorrow.

Some patients with von Willebrand disease (VWD) will experience severe bleeding complications requiring intensive treatment. Up to 40% of patients with type 3 VWD experience joint bleeding, which can lead to haemophilic arthropathy. Patients with type 2A or 2B VWD are at risk of developing recurrent gastrointestinal bleeding, which is associated with angiodysplasia. Some children with VWD will experience epistaxis of sufficient frequency, duration, or severity to cause anaemia and have negative effects on overall health, development, and quality of life. For these patients, prophylactic use of concentrates containing von Willebrand factor may help to avoid bleeding events and their associated complications. The available clinical data suggest that secondary prophylaxis is beneficial in patients with type 3 VWD and certain patients with type 1 or 2 VWD. The VWD International Prophylaxis (VIP) trial is an ongoing study that will help to identify which patients are most likely to benefit from long-term prophylaxis, and determine the optimal approach to prophylaxis in various settings.