Journal article
Validation and Reliability of the Thai Pediatric Charcot–Marie–Tooth Quality of Life Outcome Measure
Journal of the peripheral nervous system, Vol.30(4), e70075
12/2025
DOI: 10.1111/jns.70075
PMCID: PMC12620538
PMID: 41243398
Abstract
Background and Aims
Charcot–Marie–Tooth disease (CMT) is a hereditary neuropathy that causes progressive muscle weakness, sensory deficits, and impaired mobility, significantly affecting quality of life (QoL). The Pediatric Charcot–Marie–Tooth Quality of Life (pCMT-QoL) instrument was developed specifically for children with CMT. However, a validated Thai version is not yet available.
Methods
We conducted a cross-sectional study at the Pediatric Neuromuscular Clinic from July 2023 to December 2024. Using a forward–backward translation method, we adapted the pCMT-QoL into Thai. Twenty-three children with CMT and their caregivers completed the Thai questionnaire. We evaluated internal consistency using Cronbach's alpha and test–retest reliability using intraclass correlation coefficients (ICCs). Convergent validity was examined via Pearson correlation between child self-reports and parent-proxy reports across functional, mental, and physical domains.
Results
The Thai pCMT-QoL demonstrated high test–retest reliability (ICC > 0.85) and satisfactory internal consistency (Cronbach's alpha > 0.7) across most domains. Convergent validity was strong for the total and mental domains but weaker for the physical domain, reflecting differences in perception between children and parents. Parents generally reported higher QoL scores than children did, a finding consistent with studies in other neuromuscular diseases. Most participants completed the questionnaire within 15 min, suggesting good feasibility.
Interpretation
The Thai pCMT-QoL is a reliable, culturally adapted tool for assessing QoL in children with CMT. It is suitable for both remote and in-clinic administration. Future studies with larger cohorts are needed to confirm its responsiveness to clinical changes and to broaden its application in diverse settings.
Details
- Title: Subtitle
- Validation and Reliability of the Thai Pediatric Charcot–Marie–Tooth Quality of Life Outcome Measure
- Creators
- Pimchanok Kulsirichawaroj - Siriraj HospitalArisa Phochaisarn - Siriraj HospitalSivaporn Limpaninlachat - Mahidol UniversityNutchavadee Vorasan - Mahidol UniversitySurachai Likasitwattanakul - Mahidol UniversitySindhu Ramchandren - Wayne State UniversityMichael E. Shy - University of IowaOranee Sanmaneechai - Siriraj Hospital
- Resource Type
- Journal article
- Publication Details
- Journal of the peripheral nervous system, Vol.30(4), e70075
- DOI
- 10.1111/jns.70075
- PMID
- 41243398
- PMCID
- PMC12620538
- NLM abbreviation
- J Peripher Nerv Syst
- ISSN
- 1085-9489
- eISSN
- 1529-8027
- Publisher
- Wiley
- Grant note
- Siriraj research development fund, Faculty of Medicine Siriraj Hospital, Mahidol University: R016732001
This work was supported by the Siriraj research development fund, Faculty of Medicine Siriraj Hospital, Mahidol University (R016732001).
- Language
- English
- Date published
- 12/2025
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9985033872802771
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