A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients

• Published in: PloS one Vol. 12; no. 1; p. e0169878
• Main Authors: Wang, Wenjia, Guedj, Mickaël, Bertrand, Viviane, Foucquier, Julie, Jouve, Elisabeth, Commenges, Daniel, Proust-Lima, Cécile, Murphy, Niall P, Blin, Olivier, Magy, Laurent, Cohen, Daniel, Attarian, Shahram
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 01-01-2017; Public Library of Science (PLoS)
• Subjects: Adolescent; Adult; Aged; Biology and Life Sciences; Charcot-Marie-Tooth disease; Charcot-Marie-Tooth Disease - pathology; Charcot-Marie-Tooth Disease - psychology; Chi-square test; Clinical trials; Clinical Trials, Phase II as Topic; Cognitive science; Cohort Studies; Female; Humans; Item response theory; Male; Medical research; Medicine and Health Sciences; Middle Aged; Models, Theoretical; Neuropathy; Neuroscience; Outcome Assessment (Health Care); Patients; People and Places; Physical Sciences; Principal components analysis; Psychometrics; Rasch model; Research and analysis methods; Severity of Illness Index; Studies; Testing; Young Adult
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0169878

The Charcot-Marie-Tooth Neuropathy Score (CMTNS) was developed as a main efficacy endpoint for application in clinical trials of Charcot-Marie-Tooth disease type 1A (CMT1A). However, the sensitivity of the CMTNS for measuring disease severity and progression in CMT1A patients has been questioned. Here, we applied a Rasch analysis in a French cohort of patients to evaluate the psychometrical properties of the CMTNS. Overall, our analysis supports the validity of the CMTNS for application to CMT1A patients though with some limitations such as certain items of the CMTNS being more suitable for moderate to severe forms of the disease, and some items being disordered. We suggest that additional items and/or categories be considered to better assess mild-to-moderate patients.