The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome

Nicholas E Johnson; Chad Heatwole; Peter Creigh; Michael P McDermott; Nuran Dilek; Man Hung; Jerry Bounsanga; Wan Tang; Michael E Shy; David N Herrmann

doi:10.1002/ana.25282

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The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome

Journal article

Peer reviewed

The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome

Nicholas E Johnson, Chad Heatwole, Peter Creigh, Michael P McDermott, Nuran Dilek, Man Hung, Jerry Bounsanga, Wan Tang, Michael E Shy and David N Herrmann

Annals of neurology, Vol.84(2), pp.225-233

08/2018

DOI: 10.1002/ana.25282

PMCID: PMC6168418

PMID: 30014533

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https://www.ncbi.nlm.nih.gov/pmc/articles/6168418View

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Abstract

The development of a disease-specific patient-reported outcome for Charcot-Marie-Tooth disease is an important step in the preparation for therapeutic trials. This study describes the development of the Charcot-Marie-Tooth Health Index (CMTHI).\nInherited Neuropathy Consortium Contact Registry participants were queried on the symptoms that most impacted their lives. The CMTHI was developed based on these responses. Factor analysis, assessment of test-retest reliability, known group validity, and patient interviews were utilized to refine the instrument.\nThe final CMTHI contains 18 themes that capture Charcot-Marie-Tooth disease (CMT) burden. The CMTHI has a high internal consistency and test-retest reliability. The CMTHI was able to discriminate between patient groups expected to have different disease burden. The CMTHI was able to discriminate levels of disability as measured by the CMT examination score and the mobility-Disability Severity Index.\nThe CMTHI represents a valid and reliable outcome to assess patient-reported disease burden in CMT. Ann Neurol 2018;84:225-233.

Severity of Illness Index

Reproducibility of Results

Registries - standards

Humans

Middle Aged

Male

Young Adult

Adult

Female

Aged

Charcot-Marie-Tooth Disease - physiopathology

Surveys and Questionnaires - standards

Charcot-Marie-Tooth Disease - diagnosis

Patient Reported Outcome Measures

Details

Title: Subtitle: The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome
Creators: Nicholas E Johnson - Department of Neurology, Virginia Commonwealth University, Richmond, VA
Chad Heatwole - Department of Neurology, University of Rochester, Rochester, NY
Peter Creigh - Department of Neurology, University of Rochester, Rochester, NY
Michael P McDermott - Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY
Nuran Dilek - Department of Neurology, University of Rochester, Rochester, NY
Man Hung - Department of Orthopedics, University of Utah, Salt Lake City, UT
Jerry Bounsanga - Department of Orthopedics, University of Utah, Salt Lake City, UT
Wan Tang - Department of Global Biostatistics and Data Science, Tulane University, New Orleans, LA
Michael E Shy - Department of Neurology, University of Iowa, Iowa City, IA
David N Herrmann - Department of Neurology, University of Rochester, Rochester, NY
Resource Type: Journal article
Publication Details: Annals of neurology, Vol.84(2), pp.225-233
DOI: 10.1002/ana.25282
PMID: 30014533
PMCID: PMC6168418
NLM abbreviation: Ann Neurol
ISSN: 0364-5134
eISSN: 1531-8249
Publisher: United States
Grant note: K23 NS091511 / National Institute of Neurological Disorders and Stroke\nNINDS 1K23NS091511-01 / NINDS NIH HHS\nU54 NS065712 / National Institute of Neurological Disorders and Stroke\nNINDS; 1U54NS0657 / NINDS NIH HHS
Language: English
Date published: 08/2018
Academic Unit: Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
Record Identifier: 9984070700702771

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