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This article is part of the supplement: Proceedings of the Australasian Podiatry Council Conference 2011

Open Access Oral presentation

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

Joshua Burns1*, Richard Finkel2, Tim Estilow2, Andy Hiscock3, Matilde Laura4, Polly Swingle5, Agnes Patzko5, Allan Glanzman2, Gyula Acsadi6, Francesco Muntoni3, Mary Reilly4, Davide Pareyson7, Isabella Moroni7, Emanuela Pagliano7, Sindhu Ramchandren5, Kate Eichinger8, Monique Ryan9, Robert Ouvrier1, Michael Shy5 and Rosemary Shy6

Author Affiliations

1 Children's Hospital at Westmead, Sydney, Australia

2 Children's Hospital of Philadelphia, PA, USA

3 Great Ormond Street Hospital, London, UK

4 National Hospital for Neurology and Neurosurgery, London, UK

5 Wayne State University Detroit, MI, USA

6 Children's Hospital of Michigan, Detroit, MI, USA

7 C. Besta Neurological Institute, Milan, Italy

8 University of Rochester, Rochester, NY, USA

9 Royal Children's Hospital, Melbourne, Australia

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Journal of Foot and Ankle Research 2011, 4(Suppl 1):O12  doi:10.1186/1757-1146-4-S1-O12

The electronic version of this article is the complete one and can be found online at: http://www.jfootankleres.com/content/4/S1/O12


Published:20 May 2011

© 2011 Burns et al; licensee 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.

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.