Secondary Prophylaxis with Factor VIII/VWF Concentrate Sharply Reduces Bleeding in Patients with von Willebrand Disease

Long-term prophylactic administration of Octapharma’s Wilate factor VIII/von Willebrand factor(VWF) concentrate dramatically reduced bleeding episodes in 24 patients with von Willebrand disease (VWD) who were referred with a bleeding score >2 prior to diagnosis and recurrent bleeds associated with anemia despite use of on-demandVWF therapy, German hematologists reported at the International Society on Thrombosis and Hemostasis

Based on individual 24-hour post-infusion VWF:RCo recoveries, a median dose of 40 IU/kg of Wilate (range 20 to 47 IU/kg)was given on a twice weekly (15 patients), three times weekly (seven patients) or four times weekly (two patients) basis.The median duration of prophylaxis was three years.Within a 12-month period, hemoglobin levels returned to normal values in all patients.Recurrent bleeding episodes stopped in 23 of 24 patients.Over a 12-month observation period, the monthly bleeding frequency (3 vs. 0.07; p<0.001) and bleeding score (3 vs. 0, p<0.001) were significantly reduced compared with the pre-prophylaxis/ pre-diagnostic values.

The authors concluded that the use of secondary prophylactic VWF replacement therapy with Wilate is a well-tolerated, highly beneficial treatment modality for patients withVWD who present with recurrent bleeding activity despite on-demand therapy.