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Kayla M D Cornett, Manoj P Menezes, Paula Bray, Rosemary R Shy, Isabella Moroni, Emanuela Pagliano, Davide Pareyson, Tim Estilow, Sabrina W Yum, Trupti Bhandari, Francesco Muntoni, Matilde Laura, Mary M Reilly, Richard S Finkel, Katy J Eichinger, David N Herrmann, Michael E Shy, Joshua Burns
School of Health Sciences, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.
Paediatrics and Child Health, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.
Ann Clin Transl Neurol. 2020 Sep;7(9):1713-1715. doi: 10.1002/acn3.51145. Epub 2020 Aug 6.
The CMT Pediatric Scale (CMTPedS) is a reliable, valid, and responsive clinical outcome measure of disability in children with CMT. The aim of this study was to identify the most responsive patient subset(s), based on the standardized response mean (SRM), to optimize the CMTPedS as a primary outcome measure for upcoming clinical trials. Analysis was based on a 2-year natural history data from 187 children aged 3-20 years with a range of CMT genetic subtypes. Subsets based on age (3-8 years), disability level (CMTPedS score 0-14), and CMT type (CMT1A) increased the SRM of the CMTPedS considerably. Refining the inclusion criteria in clinical trials to younger, mildly affected cases of CMT1A optimizes the responsiveness of the CMTPedS.
CMT 儿科量表（CMTPedS）是一种可靠、有效且敏感的临床结局测量工具，可用于评估 CMT 患儿的残疾程度。本研究旨在根据标准化反应均值（SRM）确定最敏感的患者亚组，以优化 CMTPedS 作为即将进行的临床试验的主要结局测量指标。分析基于 187 名年龄在 3-20 岁之间的儿童的 2 年自然史数据，这些儿童患有多种 CMT 遗传亚型。基于年龄（3-8 岁）、残疾程度（CMTPedS 评分 0-14）和 CMT 类型（CMT1A）的亚组极大地提高了 CMTPedS 的 SRM。在临床试验中，将纳入标准细化为更年轻、CMT1A 病情较轻的病例，可以优化 CMTPedS 的反应性。
