Efficacy and safety of Wilate in paediatric VWD patients under 6 years of age - results of a prospective multicentre clinical study including recovery information

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2013-11, Vol.19 (6), p.887-892
• Main Authors: Nowak-Göttl, U., Krümpel, A., Russo, A., Jansen, M.
• Format: Article
• Language: English
• Subjects: Child; Child, Preschool; Coagulants - pharmacokinetics; Coagulants - therapeutic use; factor VIII; Factor VIII - therapeutic use; Female; Half-Life; Hemorrhage - prevention & control; Humans; Infant; Male; paediatric patients; Phenotype; prophylaxis; Prospective Studies; surgery; von Willebrand disease; von Willebrand Diseases - drug therapy; von Willebrand factor; von Willebrand Factor - therapeutic use
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.12237

Summary Treatment with exogenous von Willebrand factor (VWF) is indicated in patients with von Willebrand disease (VWD) in whom treatment with 1‐deamino‐8‐d‐arginine vasopressin/desmopressin is contraindicated. Wilate® is a new generation plasma‐derived concentrate of native VWF and coagulation factor VIII (FVIII) (in a physiological 1:1 ratio) developed for the treatment of VWD. This is the first study to report safety, efficacy and in vivo recovery (IVR) data from 15 paediatric patients less than 6 years of age who received Wilate® for either prophylaxis, on‐demand treatment or for treatment in surgical procedures during a prospective open‐label trial (VWD type 1: 5, type 2A: 1, type 2B: 2, type 3: 6, unknown type: 1 patients). Analysis of IVR for VWF and FVIII suggested an appropriate and consistent rise in coagulation activity after Wilate® administration. Overall efficacy was rated as excellent or good for 99.7% [prophylactic infusions] and 100% [bleeding episodes/surgical procedures]. More than 82% of bleeding episodes resolved after 1 day of treatment, and a Wilate® dosage of 20–50 IU kg−1 was sufficient to achieve haemostasis in 97% of bleeding episodes. All surgical procedures were successfully managed with Wilate®. No thromboembolic events were observed during the study, and no patient developed anti‐VWF antibodies or FVIII inhibitors. In conclusion, this study confirms both the expected IVR profile in paediatric patients and the excellent efficacy, tolerability and safety profile of Wilate® observed previously in adults. Wilate® showed excellent efficacy in the treatment of bleeding when used prophylactically or on‐demand, and in the treatment of surgical procedures.