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Validation of the parent-proxy pediatric Charcot-Marie-Tooth disease quality of life outcome measure.
Wu, Tong Tong; Finkel, Richard S; Siskind, Carly E; Feely, Shawna M E; Burns, Joshua; Reilly, Mary M; Muntoni, Francesco; Estilow, Timothy; Shy, Michael E; Ramchandren, Sindhu.
Afiliação
  • Wu TT; Department of Biostatistics and Computational Biology, University of Rochester, Rochester, New York, USA.
  • Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
  • Siskind CE; Department of Neurology, Stanford University, Stanford, California, USA.
  • Feely SME; Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA.
  • Burns J; 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.
  • Reilly MM; Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
  • Muntoni F; Centre for Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
  • Estilow T; 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.
  • Shy ME; Department of Pediatrics, Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
  • Ramchandren S; Department of Neurology, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA.
J Peripher Nerv Syst ; 28(2): 237-251, 2023 06.
Article em En | MEDLINE | ID: mdl-36748295
ABSTRACT
Charcot-Marie-Tooth disease (CMT) reduces health-related quality of life (QOL) in children. We have previously developed and validated the English and Italian versions of the pediatric CMT-specific QOL outcome measure (pCMT-QOL) for children aged 8 to 18. There is currently no parent-proxy 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-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old. Development and validation of the parent-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old was iterative, involving identifying relevant domains, item pool generation, prospective pilot testing and clinical assessments, structured focus-group 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-proxy version of the pCMT-QOL measure. From 2010 to 2016, the parent-proxy pCMT-QOL 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-retest reliability, internal consistency, convergent validity, IRT analysis, and longitudinal analysis, to develop the final parent-proxy version of the pCMT-QOL outcome measure for children aged 8 to 18 years old. The parent-proxy version of the pCMT-QOL outcome measure is a reliable, valid, and sensitive proxy measure of health-related QOL for children aged 8 to 18 with CMT.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Qualidade de Vida / Doença de Charcot-Marie-Tooth Tipo de estudo: Observational_studies / Prognostic_studies / Qualitative_research Limite: Adolescent / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Qualidade de Vida / Doença de Charcot-Marie-Tooth Tipo de estudo: Observational_studies / Prognostic_studies / Qualitative_research Limite: Adolescent / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article