Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK.
Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, UK.
Appl Health Econ Health Policy. 2023 Jul;21(4):559-584. doi: 10.1007/s40258-023-00806-8. Epub 2023 May 3.
BACKGROUND: Childhood multi-attribute utility instruments (MAUIs) can be used to measure health utilities in children (aged ≤ 18 years) for economic evaluation. Systematic review methods can generate a psychometric evidence base that informs their selection for application. Previous reviews focused on limited sets of MAUIs and psychometric properties, and only on evidence from studies that directly aimed to conduct psychometric assessments. OBJECTIVE: This study aimed to conduct a systematic review of psychometric evidence for generic childhood MAUIs and to meet three objectives: (1) create a comprehensive catalogue of evaluated psychometric evidence; (2) identify psychometric evidence gaps; and (3) summarise the psychometric assessment methods and performance by property. METHODS: A review protocol was registered with the Prospective Register of Systematic Reviews (PROSPERO; CRD42021295959); reporting followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline. The searches covered seven academic databases, and included studies that provided psychometric evidence for one or more of the following generic childhood MAUIs designed to be accompanied by a preference-based value set (any language version): 16D, 17D, AHUM, AQoL-6D, CH-6D, CHSCS-PS, CHU9D, EQ-5D-Y-3L, EQ-5D-Y-5L, HUI2, HUI3, IQI, QWB, and TANDI; used data derived from general and/or clinical childhood populations and from children and/or proxy respondents; and were published in English. The review included 'direct studies' that aimed to assess psychometric properties and 'indirect studies' that generated psychometric evidence without this explicit aim. Eighteen properties were evaluated using a four-part criteria rating developed from established standards in the literature. Data syntheses identified psychometric evidence gaps and summarised the psychometric assessment methods/results by property. RESULTS: Overall, 372 studies were included, generating a catalogue of 2153 criteria rating outputs across 14 instruments covering all properties except predictive validity. The number of outputs varied markedly by instrument and property, ranging from 1 for IQI to 623 for HUI3, and from zero for predictive validity to 500 for known-group validity. The more recently developed instruments targeting preschool children (CHSCS-PS, IQI, TANDI) have greater evidence gaps (lack of any evidence) than longer established instruments such as EQ-5D-Y, HUI2/3, and CHU9D. The gaps were prominent for reliability (test-retest, inter-proxy-rater, inter-modal, internal consistency) and proxy-child agreement. The inclusion of indirect studies (n = 209 studies; n = 900 outputs) increased the number of properties with at least one output of acceptable performance. Common methodological issues in psychometric assessment were identified, e.g., lack of reference measures to help interpret associations and changes. No instrument consistently outperformed others across all properties. CONCLUSION: This review provides comprehensive evidence on the psychometric performance of generic childhood MAUIs. It assists analysts involved in cost-effectiveness-based evaluation to select instruments based on the application-specific minimum standards of scientific rigour. The identified evidence gaps and methodological issues also motivate and inform future psychometric studies and their methods, particularly those assessing reliability, proxy-child agreement, and MAUIs targeting preschool children.
背景:儿童多属性效用量表(MAUIs)可用于测量≤18 岁儿童的健康效用,以进行经济评估。系统评价方法可以生成心理计量学证据基础,为其选择应用提供信息。以前的综述侧重于有限数量的 MAUIs 和心理计量学特性,并且仅针对直接旨在进行心理计量评估的研究的证据。
目的:本研究旨在对通用儿童 MAUIs 的心理计量学证据进行系统评价,并达到三个目的:(1)创建评估心理计量学证据的综合目录;(2)确定心理计量学证据差距;(3)总结心理计量评估方法和性能按属性。
方法:本研究方案在前瞻性系统评价注册库(PROSPERO;CRD42021295959)中进行了注册;报告遵循系统评价和荟萃分析的首选报告项目(PRISMA)2020 指南。检索涵盖了七个学术数据库,包括提供了以下一种或多种通用儿童 MAUIs 的心理计量学证据的研究,这些 MAUIs 旨在伴随偏好基础价值集(任何语言版本):16D、17D、AHUM、AQoL-6D、CH-6D、CHSCS-PS、CHU9D、EQ-5D-Y-3L、EQ-5D-Y-5L、HUI2、HUI3、IQI、QWB 和 TANDI;使用来自一般和/或临床儿童人群以及来自儿童和/或代理受访者的数据;并以英文发表。该综述包括旨在评估心理计量特性的“直接研究”和未明确此目的但产生心理计量学证据的“间接研究”。使用从文献中确立的标准制定的四部分标准评分方法对 18 个特性进行了评估。数据综合确定了心理计量学证据差距,并按属性总结了心理计量评估方法/结果。
结果:总体而言,共纳入 372 项研究,生成了 14 种工具的 2153 项标准评分输出目录,涵盖了所有特性,除了预测有效性。仪器和特性的输出数量差异很大,从 IQI 的 1 到 HUI3 的 623,以及从预测有效性的 0 到已知组有效性的 500。针对学龄前儿童的更新仪器(CHSCS-PS、IQI、TANDI)比 EQ-5D-Y、HUI2/3 和 CHU9D 等更成熟的仪器具有更大的证据差距(缺乏任何证据)。可靠性(重测、代理-评分者间、跨模式、内部一致性)和代理-儿童一致性的差距更为明显。纳入间接研究(n=209 项研究;n=900 项产出)增加了具有至少一项可接受性能产出的特性数量。心理计量评估中常见的方法学问题已确定,例如,缺乏参考措施来帮助解释关联和变化。没有一种仪器在所有特性上始终优于其他仪器。
结论:本综述提供了通用儿童 MAUIs 心理计量性能的综合证据。它帮助参与基于成本效益评估的分析师根据特定应用的科学严谨性最低标准选择仪器。确定的证据差距和方法学问题也为未来的心理计量学研究及其方法提供了动力和信息,特别是针对评估可靠性、代理-儿童一致性和针对学龄前儿童的 MAUIs 的研究及其方法。
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