Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience

Summary Children with von Willebrand disease ( VWD ) in whom DDAVP is ineffective or contraindicated require treatment with a coagulation factor concentrate containing von Willebrand factor ( VWF ) and factor VIII ( FVIII ). The aim of this study was to monitor the safety, efficacy and tolerability of Wilate ® (a VWF : FVIII concentrate with a 1:1 ratio) used across the North London Paediatric Haemophilia Network since May 2010. In total, 47 children (aged 0.0–17.0 years) with type 1 ( n  =   28), type 2 ( n  =   7), type 3 ( n  =   10) and acquired VWS ( n  =   2) have been treated for bleeds, surgery and/or prophylaxis using 260 000 IU Wilate ® . Analysis of dose and frequency of treatment show expected responses to treatment with mean doses of 55, 50 and 50 IU kg −1 for bleeds, surgery and prophylaxis respectively. Most bleeds responded to a single treatment. Surgical procedures were covered with clinician approved dosing schedules with 95% (39/41) reported as having excellent or good efficacy. There was no accumulation of FVIII or VWF and no thromboembolic events. This case series confirms the efficacy, safety and tolerability of Wilate ® in neonates, children and adolescents when used on‐demand, prophylactically and in the surgical setting.