French Multicenter Study for Post-Marketing Survey of WILFACTIN®: A Von Willebrand Factor Concentrate with a Low FVIII Content.

This non-interventional study is designed to evaluate the safety and efficacy of WILFACTIN® when used for up to 3 years of treatment of bleeding episodes at hospital, for home-self treatment or prevention of surgical bleeding by patients with von Willebrand disease (VWD). The study is set up according to the Note for guidance CPMP/BPWG/220/02. Thirty six French centers enrolled 65 patients. Data from 48 patients (26 females, 22 males) were available for this 19-month interim analysis. Patients ages ranged from 11 months to 83.5 years (median, 38.5 years): 8 had type 1, 25 type 2, and 15 type 3 VWD. Baseline VWF:RCo and FVIII:C levels were less than 20 IU/dl in 37 and 20 patients respectively. The mean observation period was 155 days. A total of 1094 infusions were administered in 39 patients, 15% for spontaneous or trauma bleeds, 23% for invasive or surgical procedures, 59% for long-term prophylaxis and 3% short-term prophylaxis. The mean infusion dosage (VWF:RCo IU/kg body weight) ranged from 35.5 to 77.7 for 10 major bleeds, 23.9 to 53.2 for 25 surgeries and 27.2 to 58.2 for 8 patients with long-term prophylaxis treatment. The majority of subjects for long-term prophylaxis had type 3 VWD (6/8) and all were more than 12 years old. Indications for prophylaxis have included mucosal bleeds for 5 patients and joint bleeds for 3 patients. The number of prophylactic infusions ranged between 1 to 3 per week and these regimens, guided by the bleeding pattern of each patient, yielded favorable results in the prevention of bleeding episodes. Only 7 bleeds occurred less than 72 hours following infusion (7/642 infusions). To date (April 2006), one patient experienced 2 non serious possibly drug-related adverse events: moderate blood pressure increase with chest pain. No cases of thrombosis were reported following treatment. No cases of VWF inhibitor development. The results of this interim analysis of post-marketing survey confirm the safety and efficacy of WILFACTIN® for treatment of VWD, particularly for home self-treatment including long-term prophylaxis, and validate the results documented in previously reported clinical trials.