{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T07:56:03Z","timestamp":1791618963829,"version":"4.3.4"},"reference-count":23,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T00:00:00Z","timestamp":1676592000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["K23\u2010NS072279"],"award-info":[{"award-number":["K23\u2010NS072279"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["U54\u2010NS065712"],"award-info":[{"award-number":["U54\u2010NS065712"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Peripheral Nervous Sys"],"published-print":{"date-parts":[[2023,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Charcot\u2010Marie\u2010Tooth disease (CMT) reduces health\u2010related quality of life (QOL) in children. We have previously developed and validated the English and Italian versions of the pediatric CMT\u2010specific QOL outcome measure (pCMT\u2010QOL) for children aged 8 to 18. There is currently no parent\u2010proxy CMT QOL outcome measure for use in clinical trials, which could provide complementary information in these children and adolescents. This study describes the validation studies conducted to develop the parent\u2010proxy version of the pCMT\u2010QOL outcome measure for children aged 8 to 18\u2009years old. Development and validation of the parent\u2010proxy version of the pCMT\u2010QOL outcome measure for children aged 8 to 18\u2009years old was iterative, involving identifying relevant domains, item pool generation, prospective pilot testing and clinical assessments, structured focus\u2010group interviews, and psychometric testing, conducted on parents of children with CMT seen at participating sites from the USA, United Kingdom, and Australia. We utilized previously described methods to develop a working parent\u2010proxy version of the pCMT\u2010QOL measure. From 2010 to 2016, the parent\u2010proxy pCMT\u2010QOL working version was administered to 358 parents of children with CMT aged 8 to 18, seen at the participating study sites of the Inherited Neuropathies Consortium. The resulting data underwent rigorous psychometric analysis, including factor analysis, test\u2010retest reliability, internal consistency, convergent validity, IRT analysis, and longitudinal analysis, to develop the final parent\u2010proxy version of the pCMT\u2010QOL outcome measure for children aged 8 to 18\u2009years old. The parent\u2010proxy version of the pCMT\u2010QOL outcome measure is a reliable, valid, and sensitive proxy measure of health\u2010related QOL for children aged 8 to 18 with CMT.<\/jats:p>","DOI":"10.1111\/jns.12538","type":"journal-article","created":{"date-parts":[[2023,2,7]],"date-time":"2023-02-07T08:46:37Z","timestamp":1675759597000},"page":"237-251","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Validation of the <scp>parent\u2010proxy<\/scp> pediatric <scp>Charcot\u2010Marie\u2010Tooth<\/scp> disease quality of life outcome measure"],"prefix":"10.1111","volume":"28","author":[{"given":"Tong Tong","family":"Wu","sequence":"first","affiliation":[{"name":"Department of Biostatistics and Computational Biology University of Rochester  Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard S.","family":"Finkel","sequence":"additional","affiliation":[{"name":"Center for Experimental Neurotherapeutics St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carly E.","family":"Siskind","sequence":"additional","affiliation":[{"name":"Department of Neurology Stanford University  Stanford California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shawna M.E.","family":"Feely","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Iowa Hospitals and Clinics  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joshua","family":"Burns","sequence":"additional","affiliation":[{"name":"Faculty of Medicine and Health; Pediatric Gait Analysis Service of New South Wales. Sydney Children's Hospitals Network University of Sydney School of Health Sciences  Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary M.","family":"Reilly","sequence":"additional","affiliation":[{"name":"Centre for Neuromuscular Diseases UCL Queen Square Institute of Neurology  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Centre for Neuromuscular Diseases UCL Queen Square Institute of Neurology  London UK"},{"name":"Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy","family":"Estilow","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Neurology Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8460-6971","authenticated-orcid":false,"given":"Michael E.","family":"Shy","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Iowa Hospitals and Clinics  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5201-9200","authenticated-orcid":false,"given":"Sindhu","family":"Ramchandren","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Michigan  Ann Arbor Michigan USA"},{"name":"Department of Neurology Wayne State University  Detroit Michigan USA"},{"name":"The Janssen Pharmaceutical Companies of Johnson &amp; 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