Why the study?
Does recombinant von Willebrand factor (rVWF) safely and effectively resolve or prevent bleeding in adults with von Willebrand disease?
Observational (n=91)
Yes
Does recombinant von Willebrand factor (rVWF) safely and effectively resolve or prevent bleeding in adults with von Willebrand disease?
Recombinant von Willebrand factor appears safe and effective for on-demand treatment of bleeds and surgical prophylaxis in adults with von Willebrand disease in a real-world setting.
rVWF may aid bleed control in von Willebrand disease; leaves open confirmation in randomized trials.
This European observational chart review assessed the efficacy/safety of recombinant von Willebrand factor (rVWF) for on-demand treatment of spontaneous/traumatic bleeds and prevention and/or treatment of surgery-related bleeding in adults with von Willebrand disease (VWD). Patients (n = 91) were enrolled at first rVWF administration (index). Data were collected for the 12 months before index and until death, loss to follow-up, or end of study (3-12 months after index). Fifteen patients reported an rVWF-treated spontaneous/traumatic bleed at index. Bleed resolution was obtained for 14 patients (unknown status, n = 1), and investigators assessed treatment satisfaction for 13 rVWF prescriptions (2 moderate, 5 good, and 6 excellent). rVWF was used to prevent/treat surgery-related bleeds at index in 76 patients. Bleed resolution was achieved in 25/58 rVWF-treated surgeries; bleed resolution was not applicable for 33 surgeries. In both groups, there were no reports of treatment-emergent adverse events after initiating rVWF, including hypersensitivity reactions, thrombotic events, and VWF inhibitor development. rVWF was shown to be effective for the on-demand treatment of spontaneous/traumatic bleeds, and for the prevention and treatment of surgical bleeds in this real-world VWD population.
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Sun et al. (2023) conducted an observational in von Willebrand disease (n=91). Recombinant von Willebrand factor (rVWF) was evaluated on Bleed resolution.
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