Boban Ana, Dubey Leonid, Vilchevska Kateryna V, Inati Adlette, Khayat Claudia Djambas, Sidonio Robert F
Department of Internal Medicine, University Hospital Centre Zagreb, Zagreb, Croatia.
Zagreb University School of Medicine, Zagreb, Croatia.
Clin Appl Thromb Hemost. 2024 Jan-Dec;30:10760296241306755. doi: 10.1177/10760296241306755.
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.
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.
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.
This post-hoc analysis demonstrated the efficacy of wilate prophylaxis in the prevention and treatment of nosebleeds in children and adults with severe VWD.
对于有频繁和严重出血史的血管性血友病（VWD）患者，建议使用血管性血友病因子（VWF）浓缩物进行预防治疗。尽管鼻出血是VWD的常见表现，但很少有研究调查因子预防在预防重度VWD患者鼻出血方面的疗效。
这项对一项为期12个月的前瞻性3期研究的事后分析，评估了Wilate在预防33例年龄≥6岁的重度1型、2型或3型VWD患者鼻出血方面的疗效。所有患者此前都参与了一项为期6个月的关于使用任何VWF浓缩物进行按需治疗的前瞻性研究。通过比较预防期间鼻出血的总年化出血率和自发年化出血率（分别为TABR和SABR）与按需治疗期间的出血率，来评估预防效果。
与按需治疗相比，预防期间鼻出血的平均TABR和SABR分别降低了76%和81%（分别为2.7对11.0以及2.1对10.9）。在预防的第二个6个月期间，平均TABR比前6个月降低了49%，平均SABR降低了57%。在按需治疗的6个月期间无鼻出血患者的比例为19%，在预防的前6个月期间为55%，在预防的第二个6个月期间为68%。Wilate治疗突破性鼻出血的疗效在99%的病例中被评为优秀。
这项事后分析证明了Wilate预防在预防和治疗重度VWD儿童和成人鼻出血方面的疗效。
