Transitioning outcome measures: relationship between the CMTPedS and CMTNSv2 in children, adolescents, and young adults with Charcot‐Marie‐Tooth disease. Issue 2 (19th June 2013)

• Record Type: Journal Article
• Authors: Burns, Joshua; Menezes, Manoj; Finkel, Richard S.; Estilow, Tim; Moroni, Isabella; Pagliano, Emanuela; Laurá, Matilde; Muntoni, Francesco; Herrmann, David N.; Eichinger, Kate; Shy, Rosemary; Pareyson, Davide; Reilly, Mary M.; Shy, Michael E.
• Is Part Of: Journal of the peripheral nervous system. Volume 18:Issue 2(2013)
• Page Start: 177
• Page End: 180
• Publication Date: 2013-06-19
• Subjects: Nervous system -- Periodicals; Nerves, Peripheral -- Diseases -- Periodicals; Peripheral Nervous System Diseases -- Periodicals; Peripheral Nervous System -- Periodicals; 612.81
• DOI: 10.1111/jns5.12024
• Languages: English
• ISSNs: 1085-9489

Abstract

<abstract abstract-type="main" id="jns512024-abs-0001"> <title>Abstract</title> <p id="jns512024-para-0001">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 &lt; 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.</p> </abstract>