Journal article
Validation of the Charcot-Marie-Tooth Disease Pediatric Scale as an Outcome Measure of Disability
Annals of neurology, Vol.71(5), pp.642-652
2012
DOI: 10.1002/ana.23572
PMID: 22522479
Abstract
Objective: Charcot-Marie-Tooth disease (CMT) is a common heritable peripheral neuropathy. There is no treatment for any form of CMT, although clinical trials are increasingly occurring. Patients usually develop symptoms during the first 2 decades of life, but there are no established outcome measures of disease severity or response to treatment. We identified a set of items that represent a range of impairment levels and conducted a series of validation studies to build a patient-centered multi-item rating scale of disability for children with CMT.
Methods: As part of the Inherited Neuropathies Consortium, patients aged 3 to 20 years with a variety of CMT types were recruited from the USA, United Kingdom, Italy, and Australia. Initial development stages involved definition of the construct, item pool generation, peer review, and pilot testing. Based on data from 172 patients, a series of validation studies were conducted, including item and factor analysis, reliability testing, Rasch modeling, and sensitivity analysis.
Results: Seven areas for measurement were identified (strength, dexterity, sensation, gait, balance, power, endurance), and a psychometrically robust 11-item scale was constructed (CMT Pediatric Scale [CMTPedS]). Rasch analysis supported the viability of the CMTPedS as a unidimensional measure of disability in children with CMT. It showed good overall model fit, no evidence of misfitting items, and no person misfit, and it was well targeted for children with CMT.
Interpretation: The CMTPedS is a well-tolerated outcome measure that can be completed in 25 minutes. It is a reliable, valid, and sensitive global measure of disability for children with CMT from the age of 3 years.
Details
- Title: Subtitle
- Validation of the Charcot-Marie-Tooth Disease Pediatric Scale as an Outcome Measure of Disability
- Creators
- Joshua BURNS - Children's Hospital at Westmead and University of Sydney, Sydney, AustraliaRobert OUVRIER - Children's Hospital at Westmead and University of Sydney, Sydney, AustraliaGyula ACSADI - Neurology Division, Connecticut Children's Medical Center, Hartford, CT, United StatesMichael E SHY - Department of Neurology, University of Iowa, Iowa City, IA, United StatesRichard S FINKEL - Departments of Neurology and Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, United StatesTim ESTILOW - Neuromuscular Program, Children's Hospital of Philadelphia, Philadelphia, PA, United StatesRosemary SHY - Department of Pediatrics, Children's Hospital of Michigan, Detroit, MI, United StatesMatilde LAURA - UCL Institute of Child Health and Great Ormond Street Hospital, London, United KingdomJulie F PALLANT - Rural Health Academic Centre, University of Melbourne, Melbourne, AustraliaMonkol LEK - Children's Hospital at Westmead and University of Sydney, Sydney, AustraliaFrancesco MUNTONI - UCL Institute of Child Health and Great Ormond Street Hospital, London, United KingdomMary M REILLY - MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology, London, United KingdomDavide PAREYSON - IRCCS Foundation, Carlo Besta Neurological Institute, Milan, Italy
- Resource Type
- Journal article
- Publication Details
- Annals of neurology, Vol.71(5), pp.642-652
- DOI
- 10.1002/ana.23572
- PMID
- 22522479
- NLM abbreviation
- Ann Neurol
- ISSN
- 0364-5134
- eISSN
- 1531-8249
- Publisher
- Wiley-Liss; Hoboken, NJ
- Language
- English
- Date published
- 2012
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984014020402771
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