Hematology Daily Report: 01/18/2024

Sidonio et al. conducted the WIL-31 trial, a phase 3 study on wilate® prophylaxis in patients with severe Von Willebrand disease (VWD) (1). The study involved 33 patients and reported a more than 50% reduction in mean total annualized bleeding rate (TABR) compared to prior on-demand treatment. The mean spontaneous annualized bleeding rate (ABR) saw an 86.9% reduction. The trial, registered under ClinicalTrials.gov identifiers NCT04053699 and NCT04052698, demonstrated the efficacy and tolerability of wilate® in pediatric and adult patients with all types of VWD.

Reference

Sidonio RF Jr, Boban A, Dubey L, Inati A, Kiss C, Boda Z, Lissitchkov TJ, Nemes L Dr, Novik D, Peteva E, Taher AT, Timofeeva MA, Vilchevska K, Vdovin V, Werner S, Knaub S, Djambas Khayat C. Von Willebrand factor/factor VIII concentrate (wilate®) prophylaxis in children and adults with von Willebrand disease. Blood Adv. 2024 Jan 18:bloodadvances.2023011742. doi: 10.1182/bloodadvances.2023011742. Epub ahead of print. PMID: 38237075.