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Title: | Efficacy of Wilate Prophylaxis in Reducing Nosebleeds in Patients with Severe VWD - A Post-hoc Analysis of the WIL-31 Study | Authors: | Boban, Ana Dubey, Leonid Vilchevska, Kateryna V Inati, Adlette Khayat, Claudia Djambas Sidonio, Robert F |
Affiliations: | Faculty of Medicine | Keywords: | Epistaxis Factor VIII Prophylaxis Von Willebrand disease Von Willebrand factor |
Issue Date: | 2024 | Publisher: | National Library of Medicine | Part of: | Clinical and Applied Thrombosis/Hemostasis | Volume: | 30 | Abstract: | Background Prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in von Willebrand disease (VWD) patients with a history of frequent and severe bleeds. Despite nosebleeds being a frequent manifestation of VWD, few studies have investigated the efficacy of factor prophylaxis in preventing nosebleeds in patients with severe VWD. Methods This post-hoc analysis of a prospective, 12-month, phase 3 study assessed the efficacy of wilate in the prevention of nosebleeds in 33 patients aged ≥6 years with severe type 1, type 2 or type 3 VWD. All patients previously participated in a 6-month prospective study of on-demand treatment with any VWF concentrate. Prophylactic efficacy was assessed by comparing total and spontaneous annualized bleeding rates for nosebleeds (TABR and SABR, respectively) during prophylaxis with rates during on-demand treatment. Results The mean TABR and SABR for nosebleeds were reduced by 76% and 81% respectively during prophylaxis versus on-demand treatment (2.7 vs 11.0 and 2.1 vs 10.9). During the second 6 months of prophylaxis, mean TABR was 49% lower and mean SABR 57% lower versus the first 6 months. The percentage of patients with zero nosebleeds was 19% during 6 months of on-demand treatment, 55% during the first 6 months of prophylaxis, and 68% during the second 6 months of prophylaxis. The efficacy of wilate in the treatment of breakthrough nosebleeds was rated excellent in 99% of cases. Conclusion This post-hoc analysis demonstrated the efficacy of wilate prophylaxis in the prevention and treatment of nosebleeds in children and adults with severe VWD. |
URI: | https://scholarhub.balamand.edu.lb/handle/uob/7792 | DOI: | 10.1177/10760296241306755 | Open URL: | Link to full text | Type: | Journal Article |
Appears in Collections: | Faculty of Medicine |
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