Qi Xingju, Zhou Wenjing, Yang Zhihao, Guannan Bai, Yang Xiaoyan, Zhang Jiaxing, Busschbach Jan
Department of Paediatrics, Guizhou Provincial People's Hospital, Guiyang, Guizhou, China.
Department of Paediatrics, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China
BMJ Open. 2025 Jun 18;15(6):e102509. doi: 10.1136/bmjopen-2025-102509.
The EQ-5D-Y (the youth version of EQ-5D) is widely used to assess children's health-related quality of life (HRQoL), yet its psychometric properties across administration modes remain insufficiently explored, particularly in paediatric oncology and rare diseases. Additionally, the broader impact of childhood illness on family caregivers (spillover effects) is underexamined. This study aims to evaluate the validity, reliability and responsiveness of the three-level version of EQ-5D-Y (EQ-5D-Y-3L) and the five-level version of EQ-5D-Y (EQ-5D-Y-5L) across different modes while also assessing the EQ-5D five-level version (EQ-5D-5L) and the new EQ Health and Well-being Short Version (EQ-HWB-9) in capturing spillover effects. Originally designed for social care interventions, the EQ-HWB-9 is expected to be applicable to caregivers.
This prospective observational study will recruit children aged 5-16 years with pneumonia, central nervous system (CNS) solid tumours or immune thrombocytopenic purpura (ITP) from three hospitals in China, along with their caregivers. A total of 360 dyads (patients and their caregivers) are planned for recruitment. Children will complete EQ-5D-Y-3L, EQ-5D-Y-5L and Paediatric Quality of Life Inventory (PedsQL) in self-complete (SC), interviewer-administered (IA) and proxy-reported modes by caregivers. Caregivers will complete EQ-5D-5L and EQ-HWB-9 to assess spillover effects. Data will be collected at baseline and follow-up (2-3 weeks). Primary outcomes include psychometric assessments (construct validity, reliability and responsiveness) of all the instruments. Secondary outcomes include HRQoL scores, ceiling effects and the correlation between EQ-5D-Y and PedsQL. A qualitative substudy will explore children's response interpretation and factors contributing to ceiling effects. Statistical analyses will include intraclass correlation coefficients for test-retest reliability, analysis of variance for known-groups validity, effect sizes for responsiveness, regression for spillover effects and thematic analysis for qualitative data.
Ethical approval has been obtained from three ethics committees: the Guizhou Medical University (IRB:2024159), the Children's Hospital, Zhejiang University School of Medicine (IRB:2024-IRB-0158-P-01) and Guizhou Provincial People's Hospital (IRB:2024073). Written informed consent will be secured from caregivers, and assent will be obtained from children aged 8 years and older. Study findings will be disseminated through national/international conferences and peer-reviewed journals.
ClinicalTrials.gov, NCT06873672.
EQ-5D-Y（EQ-5D的青少年版）被广泛用于评估儿童的健康相关生活质量（HRQoL），但其在不同施测方式下的心理测量特性仍未得到充分探索，尤其是在儿科肿瘤学和罕见病领域。此外，儿童疾病对家庭照顾者的更广泛影响（溢出效应）也未得到充分研究。本研究旨在评估三级版EQ-5D-Y（EQ-5D-Y-3L）和五级版EQ-5D-Y（EQ-5D-Y-5L）在不同施测方式下的效度、信度和反应度，同时评估EQ-5D五级版（EQ-5D-5L）和新的EQ健康与幸福简版（EQ-HWB-9）在捕捉溢出效应方面的表现。EQ-HWB-9最初是为社会护理干预设计的，预计适用于照顾者。
这项前瞻性观察性研究将从中国的三家医院招募5至16岁患有肺炎、中枢神经系统（CNS）实体瘤或免疫性血小板减少性紫癜（ITP）的儿童及其照顾者。计划招募总共360对（患者及其照顾者）。儿童将通过自我完成（SC）模式、访员施测（IA）模式以及照顾者代理报告模式完成EQ-5D-Y-3L、EQ-5D-Y-5L和儿童生活质量量表（PedsQL）。照顾者将完成EQ-5D-5L和EQ-HWB-9以评估溢出效应。数据将在基线和随访（2 - 3周）时收集。主要结局包括所有工具的心理测量评估（结构效度、信度和反应度）。次要结局包括HRQoL得分、天花板效应以及EQ-5D-Y与PedsQL之间的相关性。一项定性子研究将探索儿童对反应的解读以及导致天花板效应的因素。统计分析将包括用于重测信度的组内相关系数、用于已知组效度的方差分析、用于反应度的效应量、用于溢出效应的回归以及用于定性数据的主题分析。
已获得三个伦理委员会的伦理批准：贵州医科大学（IRB:2024159）、浙江大学医学院附属儿童医院（IRB:2024 - IRB - 0158 - P - 01）和贵州省人民医院（IRB:2024073）。将从照顾者处获得书面知情同意书，并从8岁及以上儿童处获得同意。研究结果将通过国内/国际会议和同行评审期刊进行传播。
ClinicalTrials.gov，NCT06873672。
