@article{399c443ce62b4ad9b696dd850e3b75d6,
title = "Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS",
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.",
author = "Cornett, {Kayla M.D.} and Menezes, {Manoj P.} and Paula Bray and Shy, {Rosemary R.} and Isabella Moroni and Emanuela Pagliano and Davide Pareyson and Tim Estilow and Yum, {Sabrina W.} and Trupti Bhandari and Francesco Muntoni and Matilde Laura and Reilly, {Mary M.} and Finkel, {Richard S.} and Eichinger, {Katy J.} and Herrmann, {David N.} and Shy, {Michael E.} and Joshua Burns",
note = "Funding Information: DH, FM, JB, ML, MR, MS, RF, and SY acknowledge support from the National Institutes of Neurological Diseases and Stroke and office of Rare Diseases (U54NS065712). The INC (U54NS065712) is a part of the NCATS Rare Diseases Clinical Research Network (RDCRN). MMR is grateful to the Medical Research Council (MRC), MRC Centre grant (G0601943). MMR and ML are grateful to the National Institute for Health Research University College London Hospitals Biomedical Research Centre. FM wishes to acknowledge the support of the MRC Neuromuscular Centre and of the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London and the MDUK Centre grant. JB, DP and IM acknowledge support from ACMT Rete, Charcot‐Marie‐Tooth Association and Muscular Dystrophy Association. Funding Information: DH, FM, JB, ML, MR, MS, RF, and SY acknowledge support from the National Institutes of Neurological Diseases and Stroke and office of Rare Diseases (U54NS065712). The INC (U54NS065712) is a part of the NCATS Rare Diseases Clinical Research Network (RDCRN). MMR is grateful to the Medical Research Council (MRC), MRC Centre grant (G0601943). MMR and ML are grateful to the National Institute for Health Research University College London Hospitals Biomedical Research Centre. FM wishes to acknowledge the support of the MRC Neuromuscular Centre and of the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London and the MDUK Centre grant. JB, DP and IM acknowledge support from ACMT Rete, Charcot-Marie-Tooth Association and Muscular Dystrophy Association. Publisher Copyright: {\textcopyright} 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association Copyright: Copyright 2020 Elsevier B.V., All rights reserved.",
year = "2020",
month = sep,
day = "1",
doi = "10.1002/acn3.51145",
language = "English",
volume = "7",
pages = "1713--1715",
journal = "Annals of Clinical and Translational Neurology",
issn = "2328-9503",
publisher = "Wiley-Blackwell",
number = "9",
}