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

PLoS One. 2017 Jan 17;12(1):e0169878. doi: 10.1371/journal.pone.0169878. eCollection 2017.

Abstract

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.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Charcot-Marie-Tooth Disease / pathology*
  • Charcot-Marie-Tooth Disease / psychology*
  • Clinical Trials, Phase II as Topic
  • Cohort Studies
  • Female
  • Humans
  • Male
  • Middle Aged
  • Models, Theoretical*
  • Outcome Assessment, Health Care*
  • Psychometrics*
  • Severity of Illness Index*
  • Young Adult

Grants and funding

The study was sponsored by Pharnext. The funder provided support in the form of salaries for authors WW, MG, VB, JF, NPM, DCoh, but did not have any additional role in the study design, data collection and analysis, decision to publish, or preparation of the manuscript. The specific roles of these authors are articulated in the ‘author contributions’ section.