{"title":"脊髓性肌肉萎缩症儿科患者的 EQ-5D-Y-3L、PedsQL 4.0 和 PROMIS-25 Profile v2.0 的测量特性。","authors":"Richard Huan Xu, Zuyi Zhao, Zhuxin Mao, Shengfeng Wang, Hui Xiong, Dong Dong","doi":"10.1186/s12955-024-02264-9","DOIUrl":null,"url":null,"abstract":"<p><strong>Objective: </strong>The objective of this study was to examine the psychometric properties of the EQ-5D-Y-3 L, Patient Reported Outcomes Measurement System 25-item version profile v2.0 (PROMIS-25), and Pediatric Quality of Life Inventory™ version 4.0 Generic Core Scale (PedsQL 4.0) in Chinese pediatric patients with spinal muscular atrophy (SMA).</p><p><strong>Methods: </strong>The data used in this study were obtained via a web-based cross-sectional survey. Parents of pediatric patients with SMA completed the proxy-reported EQ-5D-Y-3 L, PedsQL 4.0, and PROMIS-25 measures. Information about socioeconomic and health status was also obtained. The ceiling and floor effects, factorial structure, convergent validity, and known-group validity of the three measures were assessed.</p><p><strong>Results: </strong>Three hundred and sixty-three parents of children aged from 5 to 12 completed the questionnaires. Strong floor effects were observed for the physical function components of the PROMIS-25 (41.3%) and PedsQL 4.0 (67.8%). For EQ-5D-Y-3 L, 84.6% of the respondents reported having \"a lot of\" problems with the dimensions \"walking\" and \"looking after myself.\" Minimal ceiling or floor effects were observed for the EQ-5D-Y-3 L index value. The confirmatory factor analysis supported a six-factor structure for the PROMIS-25, but did not support a four-factor structure for the PedsQL 4.0. All hypothesized correlations of the dimensions among the three measures were confirmed, with coefficients ranging from 0.28 to 0.68. Analysis of variance showed that EQ-5D-Y-3 L demonstrated better known-group validity than the other two measures in 14 out of 16 comparisons.</p><p><strong>Conclusions: </strong>The EQ-5D-Y-3 L showed better discriminant power than the other two measures. The physical health dimensions of all three measures showed the significant floor effects. These findings provide valuable insights into the effectiveness of these measures at capturing and quantifying the impact of SMA on patients' health-related quality of life.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"50"},"PeriodicalIF":3.2000,"publicationDate":"2024-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11210123/pdf/","citationCount":"0","resultStr":"{\"title\":\"Measurement properties of the EQ-5D-Y-3L, PedsQL 4.0, and PROMIS-25 Profile v2.0 in pediatric patients with spinal muscular atrophy.\",\"authors\":\"Richard Huan Xu, Zuyi Zhao, Zhuxin Mao, Shengfeng Wang, Hui Xiong, Dong Dong\",\"doi\":\"10.1186/s12955-024-02264-9\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objective: </strong>The objective of this study was to examine the psychometric properties of the EQ-5D-Y-3 L, Patient Reported Outcomes Measurement System 25-item version profile v2.0 (PROMIS-25), and Pediatric Quality of Life Inventory™ version 4.0 Generic Core Scale (PedsQL 4.0) in Chinese pediatric patients with spinal muscular atrophy (SMA).</p><p><strong>Methods: </strong>The data used in this study were obtained via a web-based cross-sectional survey. Parents of pediatric patients with SMA completed the proxy-reported EQ-5D-Y-3 L, PedsQL 4.0, and PROMIS-25 measures. Information about socioeconomic and health status was also obtained. The ceiling and floor effects, factorial structure, convergent validity, and known-group validity of the three measures were assessed.</p><p><strong>Results: </strong>Three hundred and sixty-three parents of children aged from 5 to 12 completed the questionnaires. Strong floor effects were observed for the physical function components of the PROMIS-25 (41.3%) and PedsQL 4.0 (67.8%). For EQ-5D-Y-3 L, 84.6% of the respondents reported having \\\"a lot of\\\" problems with the dimensions \\\"walking\\\" and \\\"looking after myself.\\\" Minimal ceiling or floor effects were observed for the EQ-5D-Y-3 L index value. The confirmatory factor analysis supported a six-factor structure for the PROMIS-25, but did not support a four-factor structure for the PedsQL 4.0. All hypothesized correlations of the dimensions among the three measures were confirmed, with coefficients ranging from 0.28 to 0.68. Analysis of variance showed that EQ-5D-Y-3 L demonstrated better known-group validity than the other two measures in 14 out of 16 comparisons.</p><p><strong>Conclusions: </strong>The EQ-5D-Y-3 L showed better discriminant power than the other two measures. The physical health dimensions of all three measures showed the significant floor effects. These findings provide valuable insights into the effectiveness of these measures at capturing and quantifying the impact of SMA on patients' health-related quality of life.</p>\",\"PeriodicalId\":12980,\"journal\":{\"name\":\"Health and Quality of Life Outcomes\",\"volume\":\"22 1\",\"pages\":\"50\"},\"PeriodicalIF\":3.2000,\"publicationDate\":\"2024-06-27\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11210123/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Health and Quality of Life Outcomes\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1186/s12955-024-02264-9\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Health and Quality of Life Outcomes","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s12955-024-02264-9","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0
摘要
研究目的本研究的目的是在中国脊髓性肌萎缩症（SMA）儿科患者中研究EQ-5D-Y-3 L、患者报告结果测量系统25项版资料v2.0（PROMIS-25）和儿科生活质量量表™4.0版通用核心量表（PedsQL 4.0）的心理测量学特性：本研究通过网络横断面调查获得数据。脊髓性肌萎缩症（SMA）儿科患者的家长填写了EQ-5D-Y-3 L、PedsQL 4.0和PROMIS-25问卷。此外，还获得了有关社会经济和健康状况的信息。对这三种测量方法的上限和下限效应、因子结构、收敛有效性和已知组有效性进行了评估：有 363 名 5 至 12 岁儿童的家长填写了问卷。PROMIS-25（41.3%）和 PedsQL 4.0（67.8%）的身体功能部分均有较强的底线效应。在 EQ-5D-Y-3 L 中，84.6% 的受访者表示在 "行走 "和 "照顾自己 "方面有 "很多 "问题。EQ-5D-Y-3 L 指数值的上限或下限效应很小。确认性因子分析支持 PROMIS-25 的六因子结构，但不支持 PedsQL 4.0 的四因子结构。所有假设的三个测量维度之间的相关性都得到了证实，系数从 0.28 到 0.68 不等。方差分析显示，在 16 次比较中，EQ-5D-Y-3 L 在 14 次比较中显示出的已知组有效性优于其他两种测量方法：结论：EQ-5D-Y-3 L 的判别能力优于其他两种测量方法。结论：EQ-5D-Y-3 L 比其他两种测量方法显示出更好的区分度，所有三种测量方法的身体健康维度都显示出显著的底线效应。这些研究结果为了解这些测量方法在捕捉和量化 SMA 对患者健康相关生活质量的影响方面的有效性提供了宝贵的见解。
Measurement properties of the EQ-5D-Y-3L, PedsQL 4.0, and PROMIS-25 Profile v2.0 in pediatric patients with spinal muscular atrophy.
Objective: The objective of this study was to examine the psychometric properties of the EQ-5D-Y-3 L, Patient Reported Outcomes Measurement System 25-item version profile v2.0 (PROMIS-25), and Pediatric Quality of Life Inventory™ version 4.0 Generic Core Scale (PedsQL 4.0) in Chinese pediatric patients with spinal muscular atrophy (SMA).
Methods: The data used in this study were obtained via a web-based cross-sectional survey. Parents of pediatric patients with SMA completed the proxy-reported EQ-5D-Y-3 L, PedsQL 4.0, and PROMIS-25 measures. Information about socioeconomic and health status was also obtained. The ceiling and floor effects, factorial structure, convergent validity, and known-group validity of the three measures were assessed.
Results: Three hundred and sixty-three parents of children aged from 5 to 12 completed the questionnaires. Strong floor effects were observed for the physical function components of the PROMIS-25 (41.3%) and PedsQL 4.0 (67.8%). For EQ-5D-Y-3 L, 84.6% of the respondents reported having "a lot of" problems with the dimensions "walking" and "looking after myself." Minimal ceiling or floor effects were observed for the EQ-5D-Y-3 L index value. The confirmatory factor analysis supported a six-factor structure for the PROMIS-25, but did not support a four-factor structure for the PedsQL 4.0. All hypothesized correlations of the dimensions among the three measures were confirmed, with coefficients ranging from 0.28 to 0.68. Analysis of variance showed that EQ-5D-Y-3 L demonstrated better known-group validity than the other two measures in 14 out of 16 comparisons.
Conclusions: The EQ-5D-Y-3 L showed better discriminant power than the other two measures. The physical health dimensions of all three measures showed the significant floor effects. These findings provide valuable insights into the effectiveness of these measures at capturing and quantifying the impact of SMA on patients' health-related quality of life.
期刊介绍：
Health and Quality of Life Outcomes is an open access, peer-reviewed, journal offering high quality articles, rapid publication and wide diffusion in the public domain.
Health and Quality of Life Outcomes considers original manuscripts on the Health-Related Quality of Life (HRQOL) assessment for evaluation of medical and psychosocial interventions. It also considers approaches and studies on psychometric properties of HRQOL and patient reported outcome measures, including cultural validation of instruments if they provide information about the impact of interventions. The journal publishes study protocols and reviews summarising the present state of knowledge concerning a particular aspect of HRQOL and patient reported outcome measures. Reviews should generally follow systematic review methodology. Comments on articles and letters to the editor are welcome.
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