Prophylaxis of bleeding episodes in patients with von Willebrand's disease

Patients with severe forms of von Willebrand's disease (VWD) may have frequent haemarthroses, especially when factor VIII (FVIII) levels are below 10 U/dL, so that some of them develop target joints like patients with severe haemophilia A. Some patients have recurrent gastrointestinal bleeding, often without lesions in the gastrointestinal tract, and need treatment every day or every other day. Finally, there are children who have epistaxis frequently and severely enough to cause anaemia. In these frequent and severe bleeders, the optimal therapy may be secondary long-term prophylaxis with von Willebrand factor (VWF)/FVIII concentrates rather than on-demand treatment on the occasion of bleeding episodes. The largest experience on such prophylaxis in VWD has been in Sweden in 35 patients with severe forms of VWD. Long-term prophylaxis was also implemented in a cohort of Italian patients with VWD: prophylaxis was used in seven patients with types 3 (n = 1 ), 2A (n = 4), 2M (n = 1) and type 1 (n = 1) VWD because of recurrent gastrointestinal bleeds and in four patients with type 3 VWD because of joint bleeds. Prophylaxis prevented bleeding completely in eight patients and largely reduced hospitalisation for blood transfusions in the remaining three. The cost-effectiveness of these prophylaxis regimens versus on-demand therapy will now be investigated in one large international study