Patients with von Willebrand disease (VWD) who received prophylaxis with recombinant von Willebrand factor (rVWF) demonstrated a decrease in annualized bleed rate (ABR) compared with on-demand therapy and prophylaxis with plasma-derived VWF (pdVWF), according to the results of a nonrandomized phase 3 trial published in the journal Blood.
Long-term prophylaxis with VWF is recommended by clinical practice guidelines to prevent severe and frequent bleeding among patients with VWD. The aim of this study was to evaluate the efficacy and safety of rVWF in this population.
This open-label, nonrandomized phase 3 study treated adult patients with severe VWD who previously used on-demand therapy or prophylaxis with pdVWF with prophylactic rVWF for 12 months. The primary endpoint was ABR of spontaneous bleeding events.
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Compared with historical ABR rates of on-demand therapy, prophylactic treatment with rVWF reduced ABR by 91% (ratio, 0.085; 95% CI, 0.021-0.346). ABR was decreased by 45.0% among patients who switched from plasma-derived to rVWF (ratio, 0.550; 95% CI, 0.086-3.523).
There were 84.6% of patients who previously used on-demand treatment and 70.0% of patients who switched from pdVWF who did not report any spontaneous bleeding events during rVWF treatment.
There was 1 adverse event, which was a nonserious headache of moderate intensity, considered possibly related to rVWF. There were no reports of biding or neutralizing antibodies against rVWF or factor VIII.
The authors concluded that “rVWF prophylaxis can reduce treated spontaneous bleeding events in patients previously receiving on-demand VWF therapy and maintains at least the same level of hemostatic control in patients who switch from prophylaxis with pdVWF to rVWF, with a favorable safety profile.”
Disclosures: This study was supported by Baxalta US Inc, a Takeda company, and Baxalta Innovations GmbH, a Takeda company. Please see the original reference for a full list of disclosures.
Reference
Leebeek FWG, Peyvandi F, Escobar M, et al. Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results. Blood. 2022;140:89-98. doi: 10.1182/blood.2021014810.
