Efficacy, safety and pharmacokinetic profiles of a plasma-derived VWF/FVIII concentrate (VONCENTO®) in subjects with haemophilia A (SWIFT-HA study)

Aleksander Skotnicki, Toshko J. Lissitchkov, Vasily Mamonov, Evgeny Buevich, Kazimierz Kuliczkowski, Stefcho Goranov, Janusz Kłoczko, Anna Klukowska, Svetlana Stankovic, Liana Gercheva, Tatiana Chernova, Andrzej Hellmann, Anna Dmoszyńska, Krystyna Zawilska, Alex Veldman, Christine Joch, Wilfried Seifert

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3 Citations (Scopus)

Abstract

Introduction: VONCENTO® (CSL Behring) is a plasma-derived, high-concentration, low-volume, high-purity concentrate, which contains a high level of von Willebrand factor (VWF) high-molecular-weight multimers and a VWF/factor VIII (FVIII) ratio of ∼2.4:1, similar to Haemate® P (CSL Behring). Methods: The pharmacokinetic, efficacy and safety profiles of VONCENTO®were investigated in this multicentre, double-blind, randomised study. Subjects aged ≥12 years with haemophilia A who required treatment of nonsurgical bleeds, treatment during surgical events or who were receiving prophylaxis were included. Pharmacokineticswere investigated with a single dose of 50 IU FVIII/kg body weight of either VONCENTO® or BIOSTATE® reference product (Biostate-RP) (Day 1; Day 8 [n= 16], repeated on Day 180 [VONCENTO® only; n=15]). Efficacy and safety analyseswere performed either during on-demand treatment (n=52) or prophylaxis (n=29) for ≥6 months and ≥50 exposure days, respectively. Results: Besides the confirmation of bioequivalence between VONCENTO® and Biostate-RP, which displayed comparable PK profiles, haemostatic efficacy was rated by the investigators as either excellent or good in 96.4% of all bleeding events (96.5% spontaneous, 96.6% traumatic, 96.9% joint bleeds) as well as in 80% of major and 100% of minor surgical procedures at discharge. The median number of annualised bleeding events per subject [range] was significantly lower in the prophylaxis group (2.0 [0.0-34.6]) than in the on-demand group (14.0 [0.0-87.8], p = 0.0013).VONCENTO® was well tolerated and no inhibitory antibodies were identified during the study period. Conclusions: This study demonstrated the bioequivalence of VONCENTO® to Biostate-RP, and its excellent efficacy and safety profile in haemophilia A subjects.

Original languageEnglish
Pages (from-to)119-125
Number of pages7
JournalThrombosis Research
Volume137
DOIs
Publication statusPublished - 1 Jan 2016
Externally publishedYes

Keywords

  • Annual bleeding rate
  • Hemostatic efficacy
  • Hemostatic safety
  • On-demand therapy
  • Prophylaxis
  • Surgery

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