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

    Authors

    J. Burns; M. Menezes; R. S. Finkel; T. Estilow; I. Moroni; E. Pagliano; M. Laura; F. Muntoni; D. N. Herrmann; K. Eichinger; R. Shy; D. Pareyson; M. M. Reilly;M. E. Shy

    Comments

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    Abbreviated Journal Title

    J. Peripher. Nerv. Syst.

    Keywords

    clinical trial; inherited peripheral neuropathy; natural history; neuromuscular diseases; outcome assessment; CMT NEUROPATHY SCORE; RELIABILITY; DISABILITY; VALIDATION; Clinical Neurology; Neurosciences

    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 =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.

    Journal Title

    Journal of the Peripheral Nervous System

    Volume

    18

    Issue/Number

    2

    Publication Date

    1-1-2013

    Document Type

    Article

    Language

    English

    First Page

    177

    Last Page

    180

    WOS Identifier

    WOS:000320619400008

    ISSN

    1085-9489

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