Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience |
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| Authors: | K. Khair P. Batty R. Riat L. Bowles C. Burgess Y. ‐H. Chen D. Hart S. Platton J. Pasi R. Liesner |
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| Affiliation: | 1. Haemophilia Centre, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK;2. The Royal London Haemophilia Centre, Barts and The London School of Medicine and Dentistry, The Royal London Hospital, London, UK;3. St Bartholome w's Hospital, Haemophilia Centre, Barts and The London School of Medicine and Dentistry, The Royal London Hospital, London, UK;4. The Royal London Haemophilia Centre London, Barts and The London School of Medicine and Dentistry, QMUL, London, UK;5. Barts and The London School of Medicine and Dentistry, Haematology, The Royal London Hospital, London, UK;6. Barts and The London, Centre for Haematology ICMS, The Royal London Haemophilia Centre London, Barts and The London School of Medicine and Dentistry, QMUL, London, UK |
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| Abstract: | 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. |
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| Keywords: | bleeding children prophylaxis surgery von Willebrand disease von Willebrand factor |
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