title: A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients.
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.
authors.affiliation: Inserm U1219, Université de Bordeaux, Bordeaux, Aquitaine, France.|Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Wenjia
authors.identifier: http://orcid.org/0000-0003-0256-059X
authors.initials: W
authors.lastname: Wang
authors.affiliation: Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Mickaël
authors.initials: M
authors.lastname: Guedj
authors.affiliation: Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Viviane
authors.initials: V
authors.lastname: Bertrand
authors.affiliation: Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Julie
authors.initials: J
authors.lastname: Foucquier
authors.affiliation: CIC-CPCET, Service de Pharmacologie Clinique et Pharmacovigilance, AP-HM, Aix Marseille Université, Marseille, France.
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Jouve
authors.affiliation: Inserm U1219, Université de Bordeaux, Bordeaux, Aquitaine, France.
authors.forename: Daniel
authors.initials: D
authors.lastname: Commenges
authors.affiliation: Inserm U1219, Université de Bordeaux, Bordeaux, Aquitaine, France.
authors.forename: Cécile
authors.initials: C
authors.lastname: Proust-Lima
authors.affiliation: Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Niall P
authors.initials: NP
authors.lastname: Murphy
authors.affiliation: CIC-CPCET, Service de Pharmacologie Clinique et Pharmacovigilance, AP-HM, Aix Marseille Université, Marseille, France.
authors.forename: Olivier
authors.initials: O
authors.lastname: Blin
authors.affiliation: Centre de Référence Neuropathies Périphérique Rares, CHU de Limoges - Hôpital Dupuytren, Limoges, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Magy
authors.affiliation: Pharnext, Issy-Les-Moulineaux, France.
authors.forename: Daniel
authors.initials: D
authors.lastname: Cohen
authors.affiliation: Aix Marseille Université, INSERM, GMGF, Marseille, France.
authors.forename: Shahram
authors.initials: S
authors.lastname: Attarian
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002607:Charcot-Marie-Tooth Disease / Q000473:pathology* / Q000523:psychology*; D017322:Clinical Trials, Phase II as Topic; D015331:Cohort Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D008962:Models, Theoretical; D017063:Outcome Assessment, Health Care; D011594:Psychometrics; D012720:Severity of Illness Index; D055815:Young Adult