Development and Testing of the Psychometric Properties of 20 Bolt-On Items for the EQ-5D-5L Across 31 Rare Diseases

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health Pub Date : 2025-05-01 DOI:10.1016/j.jval.2025.01.006

Richard Huan Xu PhD , Fanni Rencz PhD , Ruiqi Sun MPhil , Dong Dong PhD , Shuyang Zhang MD

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引用次数: 0

Abstract

Objectives

Our objective was to develop and assess the psychometric properties of relevant bolt-on items for the EQ-5D-5L in patients with rare diseases (RDs).

Methods

Nineteen new EQ-5D-5L bolt-ons were developed based on literature review, expert input, and qualitative interviews and focus groups with patients, caregivers, and representatives of patient associations. A nationwide, cross-sectional, web-based survey in China included patients or caregivers of patients with 31 RDs in China (n = 9190). In each RDs, participants completed the EQ-5D-5L and 3 of 20 (1 existing and 19 newly developed) bolt-ons. Ceiling, explanatory power, convergent, divergent, and known-group validity were examined.

Results

Among the bolt-ons, itching had the lowest ceiling (6.5%), whereas social relationships had the highest (42.2%). The absolute reduction in the ceiling of the EQ-5D-5L with the addition of any bolt-ons was limited, ranging from 0 (respiratory problems) to 8.3% points (isolation). Dignity and vitality resulted in the largest increase in explained variance in EQ VAS. The isolation, fertility, and visual acuity bolt-ons showed good divergent validity from the EQ-5D-5L items. There was strong convergent validity between SF-12 and conceptually related bolt-ons (eg, physical health composite and muscle problems bolt-on). Various bolt-ons improved the known-groups validity in specific patient groups, eg, Huntington’s disease (oral expressions), scleroderma (dexterity), myasthenia gravis (muscle problems), neuromyelitis optica and multiple sclerosis (fatigue), Marfan syndrome (self-image), and Pompe disease (safety).

Conclusion

The EQ-5D-5L shows sufficient validity in most RDs, but incorporating relevant, specific bolt-ons could enhance its ability to more comprehensively assess health-related quality of life in these patients.

查看原文本刊更多论文

开发和测试了EQ-5D-5L在31种罕见疾病中的20个附加项目的心理测量特性。

目的：我们的目的是开发和评估罕见病（rd）患者EQ-5D-5L相关附加项目的心理测量特性。方法：通过文献综述、专家意见、定性访谈和对患者、护理人员和患者协会代表的焦点小组访谈，开发了19个新的EQ-5D-5L栓。在中国进行的一项全国性的、横断面的、基于网络的调查包括中国31例rd患者的患者或护理人员（n= 9190）。在每个rd中，参与者完成了EQ-5D-5L和20个（1个现有的和19个新开发的）螺栓中的3个。检验了天花板、解释力、收敛效度、发散效度和已知组效度。结果：瘙痒的上限最低（6.5%），社会关系的上限最高（42.2%）。EQ-5D-5L的上限在增加任何螺栓后的绝对降低是有限的，从0（呼吸问题）到8.3%（隔离）不等。尊严和活力导致EQ VAS解释方差增加最大。与EQ-5D-5L项目相比，隔离度、生育力和视敏度选项具有较好的差异效度。SF-12与概念相关的附加项（例如，身体健康复合和肌肉问题附加项）之间存在很强的收敛效度。在特定患者组中，各种补强可提高已知组的有效性，例如，亨廷顿氏病（口腔表达）、硬皮病（灵活性）、重症肌无力（肌肉问题）、视神经脊髓炎和多发性硬化症（疲劳）、马凡氏综合征（自我形象）和庞贝病（安全性）。结论：EQ-5D-5L在大多数rd中显示出足够的有效性，但纳入相关的特异性锚点可以增强其更全面评估这些患者健康相关生活质量的能力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Value in Health 医学-卫生保健

CiteScore

6.90

自引率

6.70%

发文量

3064

审稿时长

3-8 weeks

期刊介绍： Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.