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

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.