Journal article
Development and Validation of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Outcome Measure
Annals of neurology, Vol.89(2), pp.369-379
02/2021
DOI: 10.1002/ana.25966
PMID: 33222249
Abstract
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).
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.
We conducted systematic literature reviews and analysis of generic QOL measures to identify 6 domains relevant to QOL in children with CMT. Sixty items corresponding to those domains were developed de novo, or identified from literature review and CMT-specific modification of items from the pediatric Neuro-QOL measures. The draft version underwent prospective feasibility and face content validity assessments to develop a working version of the pCMT-QOL measure. From 2010 to 2016, the pCMT-QOL working version was administered to 398 children aged 8 to 18 years seen at the participating study sites of the Inherited Neuropathies Consortium. The resulting data underwent rigorous psychometric analysis, including factor analysis, test-retest reliability, internal consistency, convergent validity, item response theory analysis, and longitudinal analysis, to develop the final pCMT-QOL patient-reported outcome measure.
The pCMT-QOL patient-reported outcome measure is a reliable, valid, and sensitive measure of health-related QOL for children with CMT. ANN NEUROL 2021;89:369-379.
Details
- Title: Subtitle
- Development and Validation of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Outcome Measure
- Creators
- Sindhu Ramchandren - Department of Neurology, University of Michigan, Ann Arbor, MI, USAMichael E Shy - Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USATong Tong Wu - Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY, USARichard S Finkel - Division of Neurology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USACarly E Siskind - Department of Neurology, Stanford University, Stanford, CA, USAShawna M E Feely - Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USAJoshua Burns - Sydney School of Health Sciences, University of Sydney, Sydney, New South Wales, AustraliaMary M Reilly - Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, United KingdomTimothy Estilow - Division of Neurology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA
- Resource Type
- Journal article
- Publication Details
- Annals of neurology, Vol.89(2), pp.369-379
- DOI
- 10.1002/ana.25966
- PMID
- 33222249
- NLM abbreviation
- Ann Neurol
- ISSN
- 0364-5134
- eISSN
- 1531-8249
- Publisher
- United States
- Grant note
- U54NS065712 / National Center for Advancing Translation Sciences (NCATS) Rare Diseases Clinical Research Network (RDCRN) K23-NS072279 / National Institute of Neurological Disorders and Stroke (NINDS) U54-NS065712 / NINDS/ORD
- Comment
- Test Development: Pediatric Charcot–Marie–Tooth Disease Quality of Life Measure (pCMT-QOL PRO)
- Language
- English
- Date published
- 02/2021
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984070238102771
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