Efficacy, safety and consumption of plasma-derived von Willebrand factor (VWF)/Factor VIII (FVIII) concentrate with 2.4:1 VWF:FVIII ratio for the treatment of von Willebrand Disease: a systematic review

Introduction The therapeutic goal in the management of von Willebrand disease (VWD) is to treat or prevent bleeding events by correcting the deficiency of von Willebrand factor (VWF) and coagulation factor VIII (FVIII). Plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento®​/Biostate®​) is indicated in all age groups for prophylaxis and treatment of haemorrhage or surgical bleeding in patients with VWD when desmopressin is ineffective or contraindicated. A systematic literature review was conducted to evaluate the efficacy, safety and consumption of pdVWF/FVIII in the treatment of patients of all ages with mild, moderate and severe inherited VWD.

Method TheMEDLINE and Cochrane Library databases were searched for publications presenting results of studies of pdVWF/FVIII in patients with inherited VWD. All retrieved articles were assessed against predefined inclusion/exclusion criteria following Cochrane group recommendations. Pharmacovigilance data were also collected.

Results Eleven publications from eight study cohorts were identified for inclusion ([Fig. 1]). All were from multicentre studies and included both paediatric and adult patients. Eight publications included evaluations of the efficacy of pdVWF/FVIII for on-demand treatment, eight included long-term prophylactic treatment, and eight described surgical prophylaxis. Treatment protocols and pdVWF/FVIII administration methods differed between studies, as did safety evaluations. The clinical response was rated as excellent/good in 67–100% of non-surgical bleeds treated on-demand, 89–100% in the treatment of breakthrough bleeds occurring during long-term prophylaxis, while haemostatic efficacy in surgical procedures was 75–100%. Pharmacovigilance data showed low incidence rates for adverse events including FVIII/VWF inhibitors, thromboembolic events, hypersensitivity reactions and transmission of infectious agents.

Fig. 1 PRISMA flowchart of the systematic literature review.

Conclusion This review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD and treatment settings.

 

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Conflict of Interest

LR: consultant for CSL Behring and Octapharma, honoraria from LFB. GA: honoraria from CSL Behring. WT: speakers’ fees from Takeda, Bayer, Sobi, Pfizer, NovoNordisk, CSL Behring, Alexion, Portola, Sanofi and participated in advisory boards for Pfizer, Takeda, Ablynx, Sanofi, Daiichi Sankyo, LFB, Grifols and Novo Nordisk. KS and LH are employees of CSL Behring.

Publication History

Article published online:
20 February 2023