Efficacy and safety of recombinant von Willebrand factor in on-demand treatment of children with von Willebrand disease: up to 4 years of phase 3/3b follow-up

Bergmann S, Ahuja S, Albayrak C, Cnossen MH, Dunn AL, Labarque V, Luciani M, Male C, Mullins ES, Susen S, Vera PM, Mokdad AG, Wang Y, Weng J, Zhang J

A clinical study found that recombinant von Willebrand factor (rVWF) was highly effective at treating bleeding episodes in children with severe von Willebrand disease. Most bleeding events were controlled with a single infusion, and the treatment remained safe and effective for up to four years of follow-up. No serious treatment-related side effects, blood clots, or antibodies against the therapy were observed, supporting rVWF as a reliable treatment option for pediatric patients.

clinical trial; factor VIII; pediatrics; von Willebrand disease; von Willebrand factor