Transitioning outcome measures: Relationship between the CMTPedS and CMTNSv2 in children, adolescents, and young adults with Charcot-Marie-Tooth disease

Joshua Burns, Manoj Menezes, Richard S. Finkel, Tim Estilow, Isabella Moroni, Emanuela Pagliano, Matilde Laurá, Francesco Muntoni, David N. Herrmann, Kate Eichinger, Rosemary Shy, Davide Pareyson, Mary M. Reilly, Michael E. Shy

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)177-180
Number of pages4
JournalJournal of the Peripheral Nervous System
Volume18
Issue number2
DOIs
Publication statusPublished - Jun 2013

Keywords

  • clinical trial
  • inherited peripheral neuropathy
  • natural history
  • neuromuscular diseases
  • outcome assessment

ASJC Scopus subject areas

  • Clinical Neurology
  • Neuroscience(all)

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