Journal article
Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS
Annals of clinical and translational neurology, Vol.7(9), pp.1713-1715
09/2020
DOI: 10.1002/acn3.51145
PMID: 32762141
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.
Details
- Title: Subtitle
- Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS
- Creators
- Kayla M D Cornett - School of Health Sciences, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, AustraliaManoj P Menezes - Paediatrics and Child Health, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, AustraliaPaula Bray - School of Health Sciences, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, AustraliaRosemary R Shy - Department of Pediatrics, Carver College of Medicine, University of Iowa, Iowa City, IowaIsabella Moroni - Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, ItalyEmanuela Pagliano - Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, ItalyDavide Pareyson - Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, ItalyTim Estilow - Department of Occupational Therapy, The Children's Hospital of Philadelphia, Philadelphia, PennsylvaniaSabrina W Yum - Division of Neurology, Perelman School of Medicine, The Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PennsylvaniaTrupti Bhandari - UCL Institute of Child Health and National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UKFrancesco Muntoni - MRC Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London, UKMatilde Laura - MRC Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London, UKMary M Reilly - MRC Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London, UKRichard S Finkel - Translational Neurosciences (Pediatrics), St. Jude Children's Research Hospital, Memphis, TennesseeKaty J Eichinger - Department of Neurology, University of Rochester, Rochester, New YorkDavid N Herrmann - Department of Neurology, University of Rochester, Rochester, New YorkMichael E Shy - Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IowaJoshua Burns - School of Health Sciences, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, Australia
- Resource Type
- Journal article
- Publication Details
- Annals of clinical and translational neurology, Vol.7(9), pp.1713-1715
- DOI
- 10.1002/acn3.51145
- PMID
- 32762141
- NLM abbreviation
- Ann Clin Transl Neurol
- ISSN
- 2328-9503
- eISSN
- 2328-9503
- Publisher
- United States
- Language
- English
- Date published
- 09/2020
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984070663202771
Metrics
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