{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T07:55:51Z","timestamp":1791618951891,"version":"4.3.4"},"reference-count":20,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2023,5,18]],"date-time":"2023-05-18T00:00:00Z","timestamp":1684368000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["U54NS065712"],"award-info":[{"award-number":["U54NS065712"]}],"id":[{"id":"10.13039\/100000002","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":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To evaluate the parent\u2010proxy version of the pediatric Charcot Marie Tooth specific quality of life (pCMT\u2010QOL) outcome instrument for children aged 7 or younger with CMT. We have previously developed and validated the direct\u2010report pCMT\u2010QOL for children aged 8\u201318\u2009years and a parent proxy version of the instrument for children 8\u201318\u2009years old. There is currently no CMT\u2010QOL outcome measure for children aged 0\u20137\u2009years old.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Testing was conducted in parents or caregivers of children aged 0\u20137\u2009years 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\u2010proxy instrument was validated rigorously by examining previously identified domains and undergoing psychometric tests for children aged 0\u20137.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The parent\u2010proxy pCMT\u2010QOL working versions were administered to 128 parents\/caregivers of children aged 0\u20137\u2009years 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\u2010proxy version of the pCMT\u2010QOL outcome measure for children aged 0\u20137\u2009years old.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The parent\u2010proxy version of the pCMT\u2010QOL outcome measure, known as the pCMT\u2010QOL (0\u20137\u2009years parent\u2010proxy) is a valid and sensitive proxy measure of health\u2010related QOL for children aged 0\u20137\u2009years with CMT.<\/jats:p><\/jats:sec>","DOI":"10.1111\/jns.12557","type":"journal-article","created":{"date-parts":[[2023,5,11]],"date-time":"2023-05-11T14:29:54Z","timestamp":1683815394000},"page":"382-389","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Validation of the parent\u2010proxy version of the pediatric <scp>Charcot\u2010Marie\u2010Tooth<\/scp> disease quality of life instrument for children aged 0\u20137\u2009years"],"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 University of Sydney School of Health Sciences  Sydney New South Wales Australia"},{"name":"Pediatric Gait Analysis Service of New South Wales, Sydney Children's Hospitals Network  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":"Evelin","family":"Milev","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":"Division of Neurology, Department of Pediatrics 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; Johnson  Titusville New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"Childhood CMT Study Group of the Inherited Neuropathy Consortium","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,5,18]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-0004.1974.tb00638.x"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/genes5010013"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/123.7.1516"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI96499"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI159814"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1522202113"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24336"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25966"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12383"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12538"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1008810004694"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1097-0215(1999)83:12 <95::AID-IJC18>3.0.CO;2-H"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1467-842X.2000.tb00145.x"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-004-0832-9"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23572"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1529-8027.2011.00350.x"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51145"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12538"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2016.0171"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awy280"}],"container-title":["Journal of the Peripheral Nervous System"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/jns.12557","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T13:20:43Z","timestamp":1698844843000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/jns.12557"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5,18]]},"references-count":20,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,9]]}},"alternative-id":["10.1111\/jns.12557"],"URL":"https:\/\/doi.org\/10.1111\/jns.12557","archive":["Portico"],"relation":{},"ISSN":["1085-9489","1529-8027"],"issn-type":[{"value":"1085-9489","type":"print"},{"value":"1529-8027","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,5,18]]},"assertion":[{"value":"2023-03-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-05-05","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-05-18","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}