Journal article
Transitioning outcome measures: relationship between the CMTPedS and CMTNSv2 in children, adolescents, and young adults with Charcot‐Marie‐Tooth disease
Journal of the peripheral nervous system, Vol.18(2), pp.177-180
06/2013
DOI: 10.1111/jns5.12024
PMCID: PMC3714225
PMID: 23781965
Abstract
Long‐term studies of Charcot‐Marie‐Tooth (CMT) disease across the entire lifespan require stable endpoints that measure the same underlying construct (e.g., disability). The aim of this study was to assess the relationship between the CMT Pediatric Scale (CMTPedS) and the adult CMT Neuropathy Score (CMTNSv2) in 203 children, adolescents, and young adults with CMT. There was a moderate curvilinear correlation between the CMTPedS and the CMTNSv2 (Spearman's rho ρ = 0.716, p < 0.0001), although there appears to be a floor effect of the CMTNSv2 in patients with a milder CMT phenotype. Univariate analyses indicate that the relationship between the CMTPedS and CMTNSv2 scores improves with worsening disease severity and advancing age. Although one universal scale throughout life would be ideal, our data supports the transition from the CMTPedS in childhood to the CMTNSv2 in adulthood as a continuum of measuring lifelong disability in patients with CMT.
Details
- Title: Subtitle
- Transitioning outcome measures: relationship between the CMTPedS and CMTNSv2 in children, adolescents, and young adults with Charcot‐Marie‐Tooth disease
- Creators
- Joshua Burns - The Children's Hospital at WestmeadManoj Menezes - The Children's Hospital at WestmeadRichard S Finkel - Nemours Children's Hospital, University of Central Florida College of MedicineTim Estilow - The Children's Hospital of PhiladelphiaIsabella Moroni - Carlo Besta Neurological InstituteEmanuela Pagliano - Carlo Besta Neurological InstituteMatilde Laurá - UCL Institute of Child Health and Great Ormond Street HospitalFrancesco Muntoni - UCL Institute of Child Health and Great Ormond Street HospitalDavid N Herrmann - University of RochesterKate Eichinger - University of RochesterRosemary Shy - Childrens Hospital of MichiganDavide Pareyson - Carlo Besta Neurological InstituteMary M Reilly - UCL Institute of NeurologyMichael E Shy - Wayne State University School of Medicine
- Resource Type
- Journal article
- Publication Details
- Journal of the peripheral nervous system, Vol.18(2), pp.177-180
- DOI
- 10.1111/jns5.12024
- PMID
- 23781965
- PMCID
- PMC3714225
- NLM abbreviation
- J Peripher Nerv Syst
- ISSN
- 1085-9489
- eISSN
- 1529-8027
- Publisher
- Wiley Periodicals, Inc; Malden, USA
- Number of pages
- 4
- Language
- English
- Date published
- 06/2013
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984020501902771
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