title: Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience.
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.
authors.affiliation: Haemophilia Centre, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: K
authors.initials: K
authors.lastname: Khair
authors.forename: P
authors.initials: P
authors.lastname: Batty
authors.forename: R
authors.initials: R
authors.lastname: Riat
authors.forename: L
authors.initials: L
authors.lastname: Bowles
authors.forename: C
authors.initials: C
authors.lastname: Burgess
authors.forename: Y-H
authors.initials: YH
authors.lastname: Chen
authors.forename: D
authors.initials: D
authors.lastname: Hart
authors.forename: S
authors.initials: S
authors.lastname: Platton
authors.forename: J
authors.initials: J
authors.lastname: Pasi
authors.forename: R
authors.initials: R
authors.lastname: Liesner
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D019411:Clinical Laboratory Techniques; D004338:Drug Combinations; D005169:Factor VIII / Q000494:pharmacology* / Q000627:therapeutic use; D005260:Female; D006470:Hemorrhage / Q000150:complications; D006487:Hemostasis / Q000187:drug effects; D006785:Hospitals, University / Q000706:statistics & numerical data*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008131:London; D008297:Male; D012017:Referral and Consultation; D012189:Retrospective Studies; D062606:Tertiary Care Centers / Q000706:statistics & numerical data*; D014842:von Willebrand Diseases / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology / Q000601:surgery; D014841:von Willebrand Factor / Q000494:pharmacology* / Q000627:therapeutic use
chemical_list: D004338:Drug Combinations; D014841:von Willebrand Factor; D005169:Factor VIII
keywords: bleeding; children; prophylaxis; surgery; von Willebrand disease; von Willebrand factor