Work

Efficacy and safety of plasma-derived von Willebrand factor/factor VIII concentrate (wilate) prophylaxis in children and adolescents with von Willebrand disease – WIL-31 study post hoc analysis

Year: 2025

Type: article

Abstract: Prophylaxis with von Willebrand factor is recommended in people with severe von Willebrand disease (VWD), regardless of age. WIL-31, the only prospective study with an on-demand run-in study as an intraindividual comparator, demonstrated the efficacy and safety of prophylaxis with the plasma-derived von Willebrand factor/factor VIII concentrate wilate (Octapharma) in adults and children with VWD of all types. Prophylaxis is often considered in young children and adolescents with severe VWD and recurrent bleeding, although limited data support this strategy. To assess the efficacy of wilate prophylaxis in children (6-11 years) and adolescents (12-16 years) in WIL-31. Patients received 20 to 40 IU/kg (Octapharma) wilate prophylaxis 2 to 3 times weekly for 12 months. Results were compared with prospective on-demand treatment. Mean total annualized bleeding rates (ABRs) during on-demand vs prophylaxis were 32.5 vs 3.7 in children ( n = 9) and 28.9 vs 4.3 in adolescents ( n = 6), representing reductions of 89% and 85%, respectively. All 34 bleeds in children, and 20/26 (77%) bleeds in adolescents were minor. Mean spontaneous ABRs during prophylaxis were 2.5 in children and 1.5 in adolescents. The most common bleeding site in both groups and across all VWD types was the nose. ABRs were reduced further during the second 6 months of prophylaxis vs the first 6 months. During the second 6 months, 78% of children and 67% of adolescents had zero spontaneous bleeds. No serious adverse events related to study treatment or thrombotic events were observed. wilate prophylaxis was efficacious and well-tolerated in children and adolescents with all types of VWD. • Bleed prevention therapy is recommended for VWD patients of all ages. • The WIL-31 study showed that wilate helps prevent bleeds in people with all types of VWD. • wilate prophylaxis was effective in children and adolescents with VWD. • wilate prophylaxis was well tolerated in children and adolescents with VWD.

Source: Research and Practice in Thrombosis and Haemostasis

DOI: 10.1016/j.rpth.2025.102719

Authors: Robert F. Sidonio, Leonid Dubey, Kateryna V. Vilchevska, Adlette Inati, Claudia Djambas Khayat

Institutions: Emory University, University of Balamand, Saint Joseph University, Hôtel-Dieu de France

Language: English

Topic: Hemophilia Treatment and Research

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,250