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- Title
Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience.
- Authors
Khair, K.; Batty, P.; Riat, R.; Bowles, L.; Burgess, C.; Chen, Y. ‐H.; Hart, D.; Platton, S.; Pasi, J.; Liesner, R.
- 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.
- Subjects
HEMOPHILIA in children; HEMORRHAGE; VON Willebrand disease; VON Willebrand factor; BLOOD coagulation factor VIII
- Publication
Haemophilia, 2015, Vol 21, Issue 1, pe44
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.12497