In a real-world analysis of patients with inherited von Willebrand disease (VWD), recombinant von Willebrand factor (rVWF) given in conjunction with surgical procedures demonstrated safety and efficacy in preventing bleeding. Results from the analysis were reported in the journal Haemophilia.

“This study is the first to evaluate the use of rVWF in a large cohort of patients with inherited VWD in real-world conditions,” the study investigators wrote in their report. They also explained that the rVWF that they used in this study contains high-molecular-weight VWF multimers but no factor XIII (FXIII).

The study was a multicenter, retrospective analysis based in France. Patients in the study were adults with inherited VWD, and they received rVWF with surgical procedures. The study’s goals were to examine the efficacy and safety of rVWF therapy in the real-world setting. Efficacy, in terms of achievement of hemostasis, was rated using the International Society of Thrombosis and Haemostasis (ISTH) Bleeding Assessment Tool.

A total of 55 patients who underwent 63 surgical procedures were evaluated. Of these surgeries, 30 were major, and 33 were minor. The most common types of VWD were type I in 40% of patients, type 2A in 29%, and type 2M in 24%. Types 2B and 3 were represented in 3.5% of patients each.


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The total median dose of rVWF across all surgeries was 63 IU/kg (range, 12-340). For major surgeries the total median dose was 108 IU/kg (range, 22-340), and for minor surgeries it was 37 IU/kg (range, 12-288). The median number of exposure days for patients undergoing major surgeries was 3 (range, 1-14), and for those undergoing minor surgeries it was 1 (range, 1-8).

Based on ISTH criteria, clinical efficacy was considered excellent with 83% of surgeries, and it was rated as good with 14%, moderate with 1.5%, and poor with 1.5%.

FVIII:C and VWF plasma levels were evaluated during postoperative periods with a subset of surgeries (25 major and 13 minor surgeries). FVIII:C and VWF levels did not appear to accumulate over time. Adverse events reportedly did not occur either perioperatively or in the initial postoperative period. One patient died from tumor progression.

“This retrospective real-life cohort confirms the efficacy and safety of rVWF in the management of surgery to prevent bleeding in patients with all types of inherited VWD, with no accumulation either of FVIII or VWF,” the study investigators concluded in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Reference

Desprez D, Drillaud N, Flaujac C, et al. Efficacy and safety of a recombinant von Willebrand factor treatment in patients with inherited von Willebrand disease requiring surgical procedures. Haemophilia. Published online February 6, 2021. doi:10.1111/hae.14242