title: Safety and efficacy of a von Willebrand factor/factor VIII concentrate (Wilate®): a single centre experience.
abstract: von Willebrand disease (VWD) is the commonest inherited bleeding disorder. Management of major surgery or bleeding often requires treatment with a plasma-derived (pd) VWF/FVIII containing concentrate. Wilate® is a dual-virally inactivated pd-concentrate, produced specifically for the treatment of VWD, containing physiological (1:1) ratios of VWF: FVIII. We reviewed efficacy and safety of Wilate® usage (2007-2012) at our centre including 2 years following product switching the majority of patients. Clinical and laboratory data of all adult patients treated with Wilate® during the study period were evaluated. Fifty four patients used 3 972 150 IU of Wilate® (1378 infusions) between 1/3/07 and 1/5/12. Efficacy was rated as being excellent or good in 94% of surgical episodes (n = 70; 34 patients) and 98% of bleeding/traumatic episodes (n = 46; 25 patients). Eight patients (2 636 100 IU) were managed on home treatment regimens. Two patients switched to Wilate® prophylaxis in the evaluation period, demonstrating similar efficacy to a previous product. Incremental recoveries (n = 37) were 2.24 IU dL(-1) per IU kg(-1) for FVIII:C, 2.39 IU dL(-1) per IU kg(-1) for VWF:Ag and 1.88 IU dL(-1) per IU kg(-1) for VWF:RCo. Six adverse events occurred in six patients (11.1% patients) over 1378 infusions (0.44%). Half of these were retrospectively felt to be infusion speed related. No notable accumulation of FVIII was seen in patients treated for ≥3 days. There was no treatment failure, thrombosis, transfusion transmitted infection or inhibitory VWF antibodies seen. Our findings confirm safety and efficacy of Wilate® in an adult VWD population with lack of notable FVIII accumulation.
authors.affiliation: The Royal London Hospital Haemophilia Centre, Barts and The London School of Medicine and Dentistry, The Royal London Hospital, Whitechapel, London, E1 1BB, UK.
authors.forename: P
authors.initials: P
authors.lastname: Batty
authors.forename: Y-H
authors.initials: YH
authors.lastname: Chen
authors.forename: L
authors.initials: L
authors.lastname: Bowles
authors.forename: D P
authors.initials: DP
authors.lastname: Hart
authors.forename: S
authors.initials: S
authors.lastname: Platton
authors.forename: K J
authors.initials: KJ
authors.lastname: Pasi
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D004338:Drug Combinations; D005169:Factor VIII / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D008297:Male; D008875:Middle Aged; D011292:Premedication; D012189:Retrospective Studies; D013514:Surgical Procedures, Operative / Q000009:adverse effects; D016896:Treatment Outcome; D014947:Wounds and Injuries / Q000150:complications; D055815:Young Adult; D014842:von Willebrand Diseases / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D004338:Drug Combinations; C509423:factor VIII, von Willebrand factor drug combination; D014841:von Willebrand Factor; D005169:Factor VIII
keywords: bleeding; efficacy; plasma concentrate; prophylaxis; surgery; von Willebrand disease