Evidence map›Paper›PMID 41243398›Full record

ArticleJournal of the peripheral nervous system : JPNS2025

Validation and Reliability of the Thai Pediatric Charcot-Marie-Tooth Quality of Life Outcome Measure.

Pimchanok Kulsirichawaroj, Arisa Phochaisarn, Sivaporn Limpaninlachat, Nutchavadee Vorasan, Surachai Likasitwattanakul, Sindhu Ramchandren, Michael E Shy, Oranee Sanmaneechai

Abstract readValidation Study
In one paragraph

Article in Journal of the peripheral nervous system : JPNS, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

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1citing papers in PubMed
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1 · What the graph read from it

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3 · Its place in the literature

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1 citing paper in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Pimchanok KulsirichawarojDivision of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Arisa PhochaisarnDivision of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Sivaporn LimpaninlachatFaculty of Physical Therapy, Mahidol University, Bangkok, Thailand.
Nutchavadee VorasanGenetic Epidemiology Research Cluster, Research Division, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Surachai LikasitwattanakulDivision of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Sindhu RamchandrenDepartment of Neurology, Wayne State University, Detroit, Michigan, USA.ORCID https://orcid.org/0000-0002-5201-9200
Michael E ShyDepartment of Neurology, University of Iowa Hospitals and Clinics, Iowa, Iowa, USA.
Oranee SanmaneechaiDivision of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.ORCID https://orcid.org/0000-0002-4557-0387

Funding

Siriraj research development fund, Faculty of Medicine Siriraj Hospital, Mahidol University R016732001
6 · The paper itself

Abstract

BACKGROUND AND

aimsCharcot-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.

methodsWe 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.

resultsThe 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.

interpretationThe 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.

Indexed as

Charcot-Marie-Tooth DiseaseOutcome Assessment, Health CarePsychometricsQuality of LifeAdolescentChildChild, PreschoolCross-Sectional StudiesFemaleHumansMaleReproducibility of ResultsSoutheast Asian PeopleSurveys and QuestionnairesThailandCharcot–Marie–tooth diseasepatient‐reported outcome measuresquality of lifeThai pCMT‐QoLtranslation

Identifiers

PMID41243398
PMCPMC12620538

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.