Journal article
Validation of the Parent-Proxy Version of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Instrument for children aged 0-7 years
Journal of the peripheral nervous system, Vol.28(3), pp.382-389
09/2023
DOI: 10.1111/jns.12557
PMID: 37166413
Appears in UI Libraries Support Open Access
Abstract
To evaluate the parent-proxy version of the pediatric Charcot Marie Tooth specific quality of life (pCMT-QOL) outcome instrument for children aged 7 or younger with CMT. We have previously developed and validated the direct-report pCMT-QOL for children aged 8-18 years and a parent proxy version of the instrument for children 8-18 years old. There is currently no CMT-QOL outcome measure for children aged 0-7 years old.
Testing was conducted in parents or caregivers of children aged 0-7 years old with CMT evaluated at participating INC sites from the USA, United Kingdom, and Australia. The development of the instrument was iterative, involving identification of relevant domains, item pool generation, prospective pilot testing and clinical assessments, structured focus group interviews and psychometric testing. The parent-proxy instrument was validated rigorously by examining previously identified domains and undergoing psychometric tests for children aged 0-7.
The parent-proxy pCMT-QOL working versions were administered to 128 parents/caregivers of children aged 0-7 years old between 2010 and 2016. The resulting data underwent rigorous psychometric analysis, including factor analysis, internal consistency, and convergent validity, and longitudinal analysis to develop the final parent-proxy version of the pCMT-QOL outcome measure for children aged 0-7 years old.
The parent-proxy version of the pCMT-QOL outcome measure, known as the pCMT-QOL (0-7 years parent-proxy) is a valid and sensitive proxy measure of health-related QOL for children aged 0-7 years with CMT. This article is protected by copyright. All rights reserved.
Details
- Title: Subtitle
- Validation of the Parent-Proxy Version of the Pediatric Charcot-Marie-Tooth Disease Quality of Life Instrument for children aged 0-7 years
- Creators
- Tong Tong Wu - University of RochesterRichard S Finkel - St. Jude Children's Research HospitalCarly E Siskind - Stanford UniversityShawna M E Feely - Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USAJoshua BurnsMary M Reilly - National Hospital for Neurology and NeurosurgeryFrancesco Muntoni - Great Ormond Street HospitalEvelin Milev - Great Ormond Street HospitalO T Timothy Estilow - Children's Hospital of PhiladelphiaMichael E Shy - Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USASindhu Ramchandren - The Janssen Pharmaceutical Companies of Johnson & Johnson, New Jersey, USAChildhood CMT Study Group of the Inherited Neuropathy Consortium
- Resource Type
- Journal article
- Publication Details
- Journal of the peripheral nervous system, Vol.28(3), pp.382-389
- DOI
- 10.1111/jns.12557
- PMID
- 37166413
- NLM abbreviation
- J Peripher Nerv Syst
- eISSN
- 1529-8027
- Publisher
- Wiley
- Grant note
- DOI: 10.13039/100005202, name: Muscular Dystrophy Association; DOI: 10.13039/100000002, name: National Institutes of Health, award: U54NS065712
- Language
- English
- Electronic publication date
- 05/11/2023
- Date published
- 09/2023
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9984406590702771
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