Development and Validation of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Outcome Measure.

ABSTRACT:

Objective

Charcot-Marie-Tooth disease (CMT) reduces health-related quality of life (QOL), especially in children. Defining QOL in pediatric CMT can help physicians monitor disease burden clinically and in trials. We identified items pertaining to QOL in children with CMT and conducted validation studies to develop a pediatric CMT-specific QOL outcome measure (pCMT-QOL).

Methods

Development and validation of the pCMT-QOL patient-reported outcome measure were iterative, involving identifying relevant domains, item pool generation, prospective pilot testing and clinical assessments, structured focus-group interviews, and psychometric testing. Testing was conducted in children with CMT seen at participating sites from the USA, United Kingdom, and Australia.

Results

We con

SUBMITTER: Ramchandren S 

PROVIDER: S-EPMC11671102 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature