What is the efficacy of recombinant von Willebrand factor (Vonvendi) for the treatment of von Willebrand disease (vWD)?

Updated: Dec 30, 2019
  • Author: Eleanor S Pollak, MD; Chief Editor: Srikanth Nagalla, MBBS, MS, FACP  more...
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Answer

Recombinant von Willebrand factor (Vonvendi) was approved by the US Food and Drug Administration (FDA) in December 2015. Approval was based on a phase 3 trial that showed that rVWF was safe and effective in treating 192 bleeds in 22 patients with vWD and stabilized endogenous FVIII:C levels. Control of bleeding was rated good or excellent, with excellent control in 96.9% (119 of 122 minor bleeds, 59 of 61 moderate bleeds, and 6 of 7 major bleeds). A single infusion was effective in 81.8% of bleeds. [24]


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