TH Open (Apr 2024)

Exposure–Response Relationship between VWF/FVIII Activity and Spontaneous Bleeding Events Following Recombinant VWF Prophylaxis in Severe VWD

  • Frank W.G. Leebeek,
  • Giancarlo Castaman,
  • Jean François Marier,
  • Gülden Özen,
  • Indranil Bhattacharya,
  • Jingmei Zhang,
  • Scarlett Wang,
  • Yi Wang

DOI
https://doi.org/10.1055/s-0044-1787815
Journal volume & issue
Vol. 08, no. 02
pp. e243 – e251

Abstract

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Background Recombinant von Willebrand factor (rVWF, vonicog alfa, Takeda Pharmaceuticals USA) is indicated in adults diagnosed with von Willebrand disease (VWD). In this study, the exposure–response (ER) relationship between VWF activity (VWF:RCo) or factor VIII activity (FVIII:C) and spontaneous bleeding events (BEs) was evaluated in adults with severe VWD receiving rVWF prophylaxis for up to 1 year.

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