Department of Biostatistics and Computational Biology, University of Rochester, Rochester, New York, USA.
Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
J Peripher Nerv Syst. 2023 Jun;28(2):237-251. doi: 10.1111/jns.12538. Epub 2023 Feb 17.
Charcot-Marie-Tooth disease (CMT) reduces health-related quality of life (QOL) in children. We have previously developed and validated the English and Italian versions of the pediatric CMT-specific QOL outcome measure (pCMT-QOL) for children aged 8 to 18. There is currently no parent-proxy CMT QOL outcome measure for use in clinical trials, which could provide complementary information in these children and adolescents. This study describes the validation studies conducted to develop the parent-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old. Development and validation of the parent-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old was iterative, involving identifying relevant domains, item pool generation, prospective pilot testing and clinical assessments, structured focus-group interviews, and psychometric testing, conducted on parents of children with CMT seen at participating sites from the USA, United Kingdom, and Australia. We utilized previously described methods to develop a working parent-proxy version of the pCMT-QOL measure. From 2010 to 2016, the parent-proxy pCMT-QOL working version was administered to 358 parents of children with CMT aged 8 to 18, seen at the participating study sites of the Inherited Neuropathies Consortium. The resulting data underwent rigorous psychometric analysis, including factor analysis, test-retest reliability, internal consistency, convergent validity, IRT analysis, and longitudinal analysis, to develop the final parent-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old. The parent-proxy version of the pCMT-QOL outcome measure is a reliable, valid, and sensitive proxy measure of health-related QOL for children aged 8 to 18 with CMT.
Charcot-Marie-Tooth 病（CMT）降低儿童的健康相关生活质量（QOL）。我们之前已经开发并验证了适用于 8 至 18 岁儿童的英文和意大利文版儿童 CMT 特异性 QOL 结局测量量表（pCMT-QOL）。目前，在临床试验中没有用于评估父母的 CMT QOL 的量表，这可能会为这些儿童和青少年提供补充信息。本研究描述了为 8 至 18 岁儿童开发 pCMT-QOL 量表父母代理版的验证研究。开发和验证 8 至 18 岁儿童的 pCMT-QOL 量表父母代理版是迭代进行的，涉及确定相关领域、生成项目池、前瞻性试点测试和临床评估、结构化焦点小组访谈以及在来自美国、英国和澳大利亚的参与地点接受 CMT 治疗的儿童的父母中进行的心理计量学测试。我们利用先前描述的方法开发了 pCMT-QOL 量表的工作父母代理版。2010 年至 2016 年，在遗传性神经病学联合会参与研究的地点就诊的 358 名 8 至 18 岁 CMT 儿童的父母接受了 pCMT-QOL 工作父母代理版的评估。所得数据经过严格的心理计量学分析，包括因素分析、重测信度、内部一致性、收敛有效性、IRT 分析和纵向分析，以开发用于 8 至 18 岁 CMT 儿童的最终 pCMT-QOL 量表父母代理版。pCMT-QOL 量表的父母代理版是一种可靠、有效和敏感的 8 至 18 岁 CMT 儿童健康相关 QOL 的代理测量方法。
