Takeda Development Center Americas, Inc., Cambridge, MA, USA.
597121HCD Economics, Daresbury, UK.
Clin Appl Thromb Hemost. 2022 Jan-Dec;28:10760296221130334. doi: 10.1177/10760296221130334.
Recent international guidelines conditionally recommend von Willebrand factor (VWF) prophylaxis for von Willebrand disease (VWD) patients with a history of severe/frequent bleeds. This post hoc analysis of the Cost of VWD Across Europe, a Socioeconomic Study (CVESS; conducted in 2018), assessed patient characteristics and disease burden in patients aged >1 year with congenital VWD not receiving but potentially eligible for prophylaxis based on severe/frequent bleeds, and those receiving prophylaxis in the previous 12 months. Data were collected using medical records and a patient questionnaire. Patients considered potentially prophylaxis-eligible (n = 102) experienced more bleeds than patients receiving prophylaxis (n = 229) and were more likely to be admitted to the hospital due to bleeding events in the prior 12 months. Quality of life and work productivity were similar between the two groups. Logistic regression analysis showed that the prophylaxis-eligible group was more likely to have poor joint function and moderate chronic pain than the prophylaxis group. This retrospective study suggests that 1/7 patients not receiving VWF prophylaxis had a higher disease burden than patients receiving prophylaxis and would potentially benefit from prophylaxis.
最近的国际指南有条件地推荐对有严重/频繁出血史的血管性血友病（VWD）患者进行血管性血友病因子（VWF）预防治疗。本项回顾性分析（CVESS；于 2018 年开展）对欧洲血管性血友病成本，一项社会经济学研究（Cost of VWD Across Europe, a Socioeconomic Study）的资料进行了分析，评估了未接受预防治疗但根据严重/频繁出血有潜在预防治疗适应证的年龄>1 岁的先天性 VWD 患者的特征和疾病负担，以及过去 12 个月内接受预防治疗的患者的特征和疾病负担。数据是通过病历和患者问卷收集的。与接受预防治疗的患者（n = 229）相比，被认为有潜在预防治疗适应证的患者（n = 102）发生更多的出血，且更有可能因出血事件在过去 12 个月内住院。两组患者的生活质量和工作生产力相似。Logistic 回归分析显示，与预防治疗组相比，有预防治疗适应证的患者更有可能关节功能较差和有中度慢性疼痛。这项回顾性研究表明，1/7 未接受 VWF 预防治疗的患者的疾病负担比接受预防治疗的患者更高，可能从预防治疗中受益。
