Department of Biomedical Informatics, Ohio State University Wexner Medical Center, Columbus, OH.
Department of Health, Behavior and Society, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
Med Decis Making. 2021 Feb;41(2):209-221. doi: 10.1177/0272989X20978390. Epub 2021 Jan 19.
Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder with pediatric onset. Recent approvals in Duchenne have placed attention on the economic evaluation in pricing and reimbursement decisions across a range of rare conditions. We sought to assess the appropriateness of the EQ-5D, a common measure of generic health state utility, for use among patients and caregivers affected by Duchenne.
An international, cross-sectional sample of adults with Duchenne and caregivers reported patient health status using self- or proxy-reported EQ-5D-3L. Appropriateness was assessed across 6 domains of concern raised by stakeholders in Duchenne. These concerns were that the EQ-5D/EQ-VAS would not capture meaningful differences in health status, correlate with disease-specific measures, reflect real health status, exhibit face validity, be accurately interpreted, and be low burden. We evaluated these concerns by comparing EQ-5D index score and EQ-VAS scores to other condition-specific functional measures and open- and closed-ended questions.
In total, 263 participants (74% response) completed the survey, 24% of whom were adult patients. Most participants lived in the United States or United Kingdom (58%). Patient age ranged from 1 to 48 y. EQ-5D index was higher in ambulatory than nonambulatory patients (0.60 v. 0.30, < 0.001) and was negatively correlated with upper limb impairment ( = 0.61, < 0.001). Three-quarters of respondents agreed that EQ-5D measured real health status (74%). Most respondents interpreted EQ-VAS anchors of best and worst imaginable health as full health (61%) and death/near death (58%). Respondents indicated the EQ-5D was easy to understand (86%) and answer (71%).
Contrary to anecdotal concerns, we found support for the appropriateness of EQ-5D to assess health status in Duchenne. While other measures may be more sensitive to specific outcomes in Duchenne, there may be some value in results using the EQ-5D measure.
杜氏肌营养不良症是一种罕见的儿童起病的神经肌肉退行性疾病。最近在杜氏肌营养不良症方面的批准引起了人们对一系列罕见疾病的定价和报销决策中的经济评估的关注。我们试图评估 EQ-5D 的适当性，EQ-5D 是一种常用的通用健康状态效用衡量标准，适用于受杜氏肌营养不良症影响的患者和护理人员。
一项国际性、横断面的杜氏肌营养不良症成年患者及其护理人员样本通过自我或代理报告的 EQ-5D-3L 报告患者健康状况。通过在杜氏肌营养不良症利益相关者提出的 6 个关注领域评估适当性。这些问题是，EQ-5D/EQ-VAS 不会捕捉到健康状况的有意义差异，与疾病特异性措施相关，反映真实健康状况，表现出表面有效性，准确解释，并且负担低。我们通过将 EQ-5D 指数评分和 EQ-VAS 评分与其他疾病特异性功能措施以及开放和封闭式问题进行比较，来评估这些关注。
共有 263 名参与者（74%的应答率）完成了调查，其中 24%为成年患者。大多数参与者居住在美国或英国（58%）。患者年龄从 1 岁到 48 岁不等。行动能力较好的患者的 EQ-5D 指数高于行动能力较差的患者（0.60 比 0.30，<0.001），与上肢损伤呈负相关（=0.61，<0.001）。四分之三的受访者认为 EQ-5D 测量了真实的健康状况（74%）。大多数受访者将 EQ-VAS 的最佳和最差想象健康的锚点解释为完全健康（61%）和死亡/接近死亡（58%）。受访者表示 EQ-5D 易于理解（86%）和回答（71%）。
与传闻中的担忧相反，我们发现 EQ-5D 适用于评估杜氏肌营养不良症的健康状况。虽然其他措施可能对杜氏肌营养不良症的特定结果更敏感，但使用 EQ-5D 测量可能具有一定的价值。
Zotero 插件