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Gepubliceerd in: Journal of Foot and Ankle Research 1/2011

Open Access 01-12-2011 | Oral presentation

Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS)

Auteurs: Joshua Burns, Richard Finkel, Tim Estilow, Andy Hiscock, Matilde Laura, Polly Swingle, Agnes Patzko, Allan Glanzman, Gyula Acsadi, Francesco Muntoni, Mary Reilly, Davide Pareyson, Isabella Moroni, Emanuela Pagliano, Sindhu Ramchandren, Kate Eichinger, Monique Ryan, Robert Ouvrier, Michael Shy, Rosemary Shy

Gepubliceerd in: Journal of Foot and Ankle Research | bijlage 1/2011

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Background

Charcot-Marie-Tooth disease (CMT) causes peripheral nerve demyelination, progressive foot weakness, cavus deformity, difficulty walking and sensory loss. There is a need for accurate, sensitive and disease-relevant measures of young children through to adolescents with CMT to enable accurate assessment of baseline performance, monitor disease severity longitudinally, and determine responses to existing and novel foot and ankle interventions. Our objective was to develop a multidimensional scale to measure disease severity of children with CMT, known as the CMT Pediatric Scale (CMTPedS).

Methods

The CMTPedS has undergone a thorough development process: (1) definition of the construct; (2) generation of the item pool; (3) choice of scoring format; (4) peer-review (face validity); (5) pilot testing; (6) standardised training; (7) inter-rater reliability of four international centres assessing eight children with CMT; (8) multicenter implementation.

Results

Findings of the development process: (1) the CMTPedS is a composite scale with broad application to measure disease severity of childhood CMT with eight domains capturing symptoms, foot/ankle involvement, lower limb sensation, hand dexterity/strength, balance, motor function; (2) a large pool of items generated from the literature were reduced based on disease-specificity, functional/patient-relevance, reliability/validity, published norms, test duration and ease of interpretation; (3) items collapsed to 5-point Likert scales using z-scores based on age/gender norms; (4) quality, appropriateness and suitability of items peer-reviewed by 23 expert clinicians/researchers/patient representatives at the 168th European Neuromuscular Centre International Workshop; (5) pilot-tested on four children with CMT to check for administration problems, item instructions, order and duration; (6) clinicians from USA, UK, Italy and Australia trained through workshops, online manual and video resources; (7) all items exhibited good to excellent inter-rater reliability (ICC2,40.78-0.99) (8) a multicenter natural history study of children with all types of CMT aged 3-17 years is underway, with 90 children recruited to date.

Conclusions

Application and psychometric validation of the CMTPedS continues. We plan to apply the final CMTPedS as the primary outcome in clinical trials of podiatric, pharmacological and surgical interventions.
This article is published under license to BioMed Central Ltd. This is an open access article distributed under the terms of the Creative Commons Attribution License (http://​creativecommons.​org/​licenses/​by/​2.​0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
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Metagegevens
Titel
Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS)
Auteurs
Joshua Burns
Richard Finkel
Tim Estilow
Andy Hiscock
Matilde Laura
Polly Swingle
Agnes Patzko
Allan Glanzman
Gyula Acsadi
Francesco Muntoni
Mary Reilly
Davide Pareyson
Isabella Moroni
Emanuela Pagliano
Sindhu Ramchandren
Kate Eichinger
Monique Ryan
Robert Ouvrier
Michael Shy
Rosemary Shy
Publicatiedatum
01-12-2011
Uitgeverij
BioMed Central
Gepubliceerd in
Journal of Foot and Ankle Research / Uitgave bijlage 1/2011
Elektronisch ISSN: 1757-1146
DOI
https://doi.org/10.1186/1757-1146-4-S1-O12