Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS)
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ORAL PRESENTATION Open Access Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS) Joshua Burns 1* , Richard Finkel 2 , Tim Estilow 2 , Andy Hiscock 3 , Matilde Laura 4 , Polly Swingle 5 , Agnes Patzko 5 , Allan Glanzman 2 , Gyula Acsadi 6 , Francesco Muntoni 3 , Mary Reilly 4 , Davide Pareyson 7 , Isabella Moroni 7 , Emanuela Pagliano 7 , Sindhu Ramchandren 5 , Kate Eichinger 8 , Monique Ryan 9 , Robert Ouvrier 1 , Michael Shy 5 , Rosemary Shy 6 From Australasian Podiatry Council Conference 2011 Melbourne, Australia. 26-29 April 2011 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 mea- sures of young children through to adolescents with CMT to enable accurate assessment of baseline perfor- mance, 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) multicen- ter 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 func- tion; (2) a large pool of items generated from the litera- ture were reduced based on disease-specificity, functional/patient-relevance, reliability/validity, pub- lished 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, appro- priateness and suitability of items peer-reviewed by 23 expert clinicians/researchers/patient representatives at the 168th European Neuromuscular Centre Interna- tional 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 work- shops, online manual and video resources; (7) all items exhibited good to excellent inter-rater reliability (ICC 2,4 0.78-0.99) (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. Author details 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. * Correspondence: [email protected] 1 Children’s Hospital at Westmead, Sydney, Australia Full list of author information is available at the end of the article Burns et al. Journal of Foot and Ankle Research 2011, 4(Suppl 1):O12 http://www.jfootankleres.com/content/4/S1/O12 JOURNAL OF FOOT AND ANKLE RESEARCH © 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.
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Transcript of Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS)
ORAL PRESENTATION Open Access
Development, reliability and validity of theCharcot-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,Rosemary Shy6
From Australasian Podiatry Council Conference 2011Melbourne, Australia. 26-29 April 2011
BackgroundCharcot-Marie-Tooth disease (CMT) causes peripheralnerve demyelination, progressive foot weakness, cavusdeformity, difficulty walking and sensory loss. There is aneed for accurate, sensitive and disease-relevant mea-sures of young children through to adolescents withCMT to enable accurate assessment of baseline perfor-mance, monitor disease severity longitudinally, anddetermine responses to existing and novel foot andankle interventions. Our objective was to develop amultidimensional scale to measure disease severity ofchildren with CMT, known as the CMT Pediatric Scale(CMTPedS).
MethodsThe CMTPedS has undergone a thorough developmentprocess: (1) definition of the construct; (2) generation ofthe item pool; (3) choice of scoring format; (4) peer-review (face validity); (5) pilot testing; (6) standardisedtraining; (7) inter-rater reliability of four internationalcentres assessing eight children with CMT; (8) multicen-ter implementation.
ResultsFindings of the development process: (1) the CMTPedSis a composite scale with broad application to measuredisease severity of childhood CMT with eight domainscapturing symptoms, foot/ankle involvement, lower limb
sensation, hand dexterity/strength, balance, motor func-tion; (2) a large pool of items generated from the litera-ture were reduced based on disease-specificity,functional/patient-relevance, reliability/validity, pub-lished norms, test duration and ease of interpretation;(3) items collapsed to 5-point Likert scales usingz-scores based on age/gender norms; (4) quality, appro-priateness and suitability of items peer-reviewed by 23expert clinicians/researchers/patient representatives atthe 168th European Neuromuscular Centre Interna-tional Workshop; (5) pilot-tested on four children withCMT to check for administration problems, iteminstructions, order and duration; (6) clinicians fromUSA, UK, Italy and Australia trained through work-shops, online manual and video resources; (7) all itemsexhibited good to excellent inter-rater reliability(ICC2,40.78-0.99) (8) a multicenter natural history studyof children with all types of CMT aged 3-17 years isunderway, with 90 children recruited to date.
ConclusionsApplication and psychometric validation of theCMTPedS continues. We plan to apply the finalCMTPedS as the primary outcome in clinical trials ofpodiatric, pharmacological and surgical interventions.
Author details1Children’s Hospital at Westmead, Sydney, Australia. 2Children’s Hospital ofPhiladelphia, PA, USA. 3Great Ormond Street Hospital, London, UK. 4NationalHospital for Neurology and Neurosurgery, London, UK. 5Wayne StateUniversity Detroit, MI, USA. 6Children’s Hospital of Michigan, Detroit, MI, USA.* Correspondence: [email protected]
1Children’s Hospital at Westmead, Sydney, AustraliaFull list of author information is available at the end of the article
Burns et al. Journal of Foot and Ankle Research 2011, 4(Suppl 1):O12http://www.jfootankleres.com/content/4/S1/O12
JOURNAL OF FOOTAND ANKLE RESEARCH
© 2011 Burns et al; licensee BioMed Central Ltd. This is an open access article distributed under the terms of the Creative CommonsAttribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction inany medium, provided the original work is properly cited.
7C. Besta Neurological Institute, Milan, Italy. 8University of Rochester,Rochester, NY, USA. 9Royal Children’s Hospital, Melbourne, Australia.
Published: 20 May 2011
doi:10.1186/1757-1146-4-S1-O12Cite this article as: Burns et al.: Development, reliability and validity ofthe Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS). Journal ofFoot and Ankle Research 2011 4(Suppl 1):O12.
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Burns et al. Journal of Foot and Ankle Research 2011, 4(Suppl 1):O12http://www.jfootankleres.com/content/4/S1/O12
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