Health Economics Unit, Melbourne School of Population and Global Health, University of Melbourne, 4/207 Bouverie Street, Carlton, VIC, 3053, Australia.
National Perinatal Epidemiology Unit, Nuffield Department of Population Health, University of Oxford, Oxford, UK.
Pharmacoeconomics. 2024 Jun;42(Suppl 1):129-145. doi: 10.1007/s40273-024-01350-6. Epub 2024 Jan 18.
Few preference-weighted instruments are available to measure health-related quality of life in young children (2-4 years of age). The EQ-5D-Y-3L and EQ-5D-Y-5L were recently modified for this purpose.
The aim of this study was to test the psychometric properties of these adapted versions for use with parent proxies of children aged 2-4 years and to compare their performance with the original versions. It was hypothesised that the adapted instrument wording would result in improved psychometric performance.
Survey data of children aged 2-4 years were obtained from the Australian Paediatric Multi-Instrument Comparison study. Distributional and psychometric properties tested included feasibility, convergence, distribution of level scores, ceiling effects, known-group validity (Cohen's D effect sizes for prespecified groups defined by the presence/absence of special healthcare needs [SHCNs]), test-retest reliability (intraclass correlation coefficients [ICCs]), and responsiveness (standardised response mean [SRM] effect sizes for changes in health). Level sum scores were used to provide summary outcomes. Supplementary analysis using utility scores (from the Swedish EQ-5D-Y-3L value set) were conducted for the adapted and original EQ-5D-Y-3L, and no value sets were available for the EQ-5D-Y-5L.
A total of 842 parents of children aged 2-4 years completed the survey. All instruments were easy to complete. There was strong convergence between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed more responses in the severe levels of the five EQ-5D-Y dimensions, particularly in the usual activity and mobility dimensions (EQ-5D-Y-5L: mobility level 1: adapted n = 478 [83%], original n = 253 [94%]; mobility level 4/5: adapted n = 17 [2.9%], original n = 4 [1.5%)]). The difference in the distribution of responses was more evident in children with SHCNs. Assessment of known-group validity showed a greater effect size for the adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L compared with the original instruments (EQ-5D-Y-5L: adapted Cohen's D = 1.01, original Cohen's D = 0.83) between children with and without SHCNs. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed improved reliability at 4-week follow-up, with improved ICCs (EQ-5D-Y-5L: adapted ICC = 0.83, original ICC = 0.44). The responsiveness of all instruments moved in the hypothesised direction for better or worse health at follow-up. Probability of superiority analysis showed little/no differences between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. Supplementary psychometric analysis of the adapted and original EQ-5D-Y-3L using utilities showed comparable findings with analyses using level sum scores.
The findings suggest improved psychometric performance of the adapted version of the EQ-5D-Y-3L and EQ-5D-Y-5L in children aged 2-4 years compared with the original versions.
目前仅有少数偏好加权工具可用于衡量 2-4 岁幼儿的健康相关生活质量。最近对 EQ-5D-Y-3L 和 EQ-5D-Y-5L 进行了修改以实现这一目标。
本研究旨在测试这两种经过改编的版本在 2-4 岁儿童的父母代理使用中的心理测量性能，并与原始版本进行比较。假设改编后的仪器措辞将导致心理测量性能的提高。
从澳大利亚儿科多仪器比较研究中获得了 2-4 岁儿童的调查数据。测试的分布和心理测量特性包括可行性、收敛性、水平得分分布、天花板效应、已知组有效性（根据特殊医疗需求 [SHCNs] 的存在/不存在定义的特定组的 Cohen's D 效应大小）、测试-再测试可靠性（ICC）和响应能力（健康变化的标准化反应均值 [SRM] 效应大小）。使用等级总和得分提供汇总结果。使用效用得分（来自瑞典 EQ-5D-Y-3L 值集）对经过改编和原始 EQ-5D-Y-3L 进行了补充分析，而 EQ-5D-Y-5L 则没有值集。
共有 842 名 2-4 岁儿童的父母完成了调查。所有仪器都易于完成。改编后的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 与原始 EQ-5D-Y-3L 和 EQ-5D-Y-5L 之间具有很强的收敛性。经过改编的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 在五个 EQ-5D-Y 维度的严重程度上表现出更多的反应，特别是在日常活动和移动性维度上（EQ-5D-Y-5L：移动性水平 1：改编 n = 478 [83%]，原始 n = 253 [94%]；移动性水平 4/5：改编 n = 17 [2.9%]，原始 n = 4 [1.5%]）。在有 SHCNs 的儿童中，反应分布的差异更为明显。对已知组有效性的评估表明，与原始仪器相比，经过改编的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 的效果更大（EQ-5D-Y-5L：改编后的 Cohen's D = 1.01，原始 Cohen's D = 0.83）在有和没有 SHCNs 的儿童之间。经过改编的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 在 4 周随访时具有更好的可靠性，改进后的 ICC 更高（EQ-5D-Y-5L：改编后的 ICC = 0.83，原始 ICC = 0.44）。所有仪器的反应能力在随访时朝着更好或更差的健康方向发展。优势概率分析表明，经过改编的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 与原始版本之间几乎没有差异。使用效用对经过改编和原始 EQ-5D-Y-3L 进行的补充心理测量分析显示，与使用等级总和得分进行的分析结果相似。
研究结果表明，与原始版本相比，2-4 岁儿童的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 经过改编的版本在心理测量性能方面有了提高。
