Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease: A Systematic Review and Pharmacovigilance Update.

Abstract

Background: Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management.

Methods: This systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982-31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria. Pharmacovigilance data were also retrieved for the same period.

Results: Fifteen studies were identified, 12 being observational and three interventional. Efficacy and safety assessments and treatment protocols varied across the studies which hindered direct comparisons. Haemostatic efficacy of pdVWF/FVIII was rated excellent/good for OD treatment in 95%-98% of bleeds and in 94%-100% of surgeries. In two separate studies, prophylactic efficacy was rated excellent/good in 100% of treatment cycles. Where reported, median annualized bleeding rates decreased from 3-24 prior prophylaxis to 0.5-6 during prophylaxis. Analysis of pharmacovigilance safety reports showed that pdVWF/FVIII was associated with a low rate of adverse events.

Conclusions: This systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD.