학술논문
Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS.
Document Type
Article
Author
Cornett, Kayla M. D.; Menezes, Manoj P.; Bray, Paula; Shy, Rosemary R.; Moroni, Isabella; Pagliano, Emanuela; Pareyson, Davide; Estilow, Tim; Yum, Sabrina W.; Bhandari, Trupti; Muntoni, Francesco; Laura, Matilde; Reilly, Mary M.; Finkel, Richard S.; Eichinger, Katy J.; Herrmann, David N.; Shy, Michael E.; Burns, Joshua
Source
Subject
*CLINICAL trials
*CHILDREN with disabilities
*PATIENT selection
*HEALTH status indicators
*PROGNOSIS
*CHARCOT-Marie-Tooth disease
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Language
ISSN
2328-9503
Abstract
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. [ABSTRACT FROM AUTHOR]