Abstract
Validation of the Charcot-Marie-Tooth Pediatric Scale as an Outcome Measure of Disability (P05.142)
Neurology, Vol.78(Meeting Abstracts 1), pp.P05-P05.142
04/25/2012
DOI: 10.1212/WNL.78.1_MeetingAbstracts.P05.142
Abstract
Objective: To develop and validate a scoring instrument that will measure impairment and progression in children with inherited neuropathy.
Background Charcot-Marie-Tooth disease (CMT) consists of a common group of heritable peripheral neuropathies. There is no treatment for any form of CMT although clinical trials are increasingly occurring. Patients usually develop symptoms during the first two decades of life but there are no established 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 in children with CMT.
Design/Methods: As part of the Inherited Neuropathies Consortium, patients aged 3-20 years with a variety of CMT types were recruited from the USA, UK, Italy and Australia. Initial development stages involved: definition of the construct, generation of the item pool, peer review and pilot testing. Based on data from 172 patients, a series of validation studies were conducted: item analysis, factor analysis, reliability testing, Rasch modeling, sensitivity analysis and responsiveness testing. A worldwide implementation procedure was also developed.
Results: Seven areas for measurement were identified (dexterity, strength, sensation, gait, balance, power, endurance), and a psychometrically robust 11-item scale constructed (Charcot-Marie-Tooth disease Pediatric Scale: CMTPedS). Rasch analysis supported the viability of the CMTPedS as a unidimensional measure of disability in children with CMT.
Conclusions: The CMTPedS is a reliable, valid and sensitive disease-relevant measure for children and young adults with CMT. As such, the CMTPedS may serve as an effective primary outcome measure to enable precise assessment of baseline performance, monitor disease severity longitudinally, and determine responses to existing and novel interventions.
Details
- Title: Subtitle
- Validation of the Charcot-Marie-Tooth Pediatric Scale as an Outcome Measure of Disability (P05.142)
- Creators
- M Shy - University of Iowa, NeurologyR OuvrierT EstilowM LauraJ PallantM LekE PaglianoI MoroniR Shy - University of Iowa, NeurologyM ReillyD PareysonF MuntoniR FinkelJ Burns
- Resource Type
- Abstract
- Publication Details
- Neurology, Vol.78(Meeting Abstracts 1), pp.P05-P05.142
- DOI
- 10.1212/WNL.78.1_MeetingAbstracts.P05.142
- ISSN
- 0028-3878
- eISSN
- 1526-632X
- Language
- English
- Date published
- 04/25/2012
- Academic Unit
- Molecular Physiology and Biophysics; Neurology; Iowa Neuroscience Institute; Stead Family Department of Pediatrics
- Record Identifier
- 9984072098202771
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