ArticleAnnals of neurology2018
The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome.
Article in Annals of neurology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
27 citing papers in PubMed.
- A phase III clinical trial of PXT3003 in Chinese patients with Charcot-Marie-Tooth disease type 1A.iScience · 2026Article
- Clinical Development of Therapies for Charcot-Marie-Tooth Disease: Recommendations for Trial Design, Endpoints, and Regulatory Pathways.Journal of the peripheral nervous system : JPNS · 2026Review
- Development and Validation of a Novel, Disease-Specific, Caregiver-Reported Outcome Measure: The Duchenne Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI).Muscle & nerve · 2026Article
- Fatigue as a modifier of diet quality in adults with Charcot Marie Tooth disease.Journal of neuromuscular diseases · 2026Article
- Development and Validation of the Inclusion Body Myositis-Health Index: A Disease-Specific Patient-Reported Outcome Measure for Use in Clinical Trials and Drug-Labeling Claims.Journal of clinical neuromuscular disease · 2026Article
- The Parkinson's Disease-Health Index: A novel, disease-specific patient-reported outcome measure.Journal of Parkinson's disease · 2026Article
- Development and Validation of a Visual Grading Score of Disease Severity From Gait Videos in Genetic Peripheral Neuropathy.European journal of neurology · 2026Article
- Charcot-Marie-Tooth disease and related neuropathies.Nature reviews. Disease primers · 2026Review
- Feasibility, Validity, and Reliability of the Virtual CMT Infant Toddler Scale (vCMTInfS): A Remote Evaluation of Infants/Toddlers With CMT.Journal of the peripheral nervous system : JPNS · 2025Article
- All hands on deck: The multidisciplinary rehabilitation assessment and management of hand function in persons with neuromuscular disorders.Muscle & nerve · 2025Review
- Review
- Patient-reported disease burden in the Accelerate Clinical Trials in Charcot-Marie-Tooth Disease Study.Journal of the peripheral nervous system : JPNS · 2024Article
- Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.Neurology. Clinical practice · 2024Article
- The Limb Girdle Muscular Dystrophy Health Index (LGMD-HI).Neuromuscular disorders : NMD · 2024Article
- Article
- Clinical and Genetic Aspects of Childhood-Onset Demyelinating Charcot-Marie-Tooth's Disease in Brazil.Journal of pediatric genetics · 2023Article
- Friedreich's Ataxia-Health Index: Development and Validation of a Novel Disease-Specific Patient-Reported Outcome Measure.Neurology. Clinical practice · 2023Article
- Response rates in clinical quality registries and databases that collect patient reported outcome measures: a scoping review.Health and quality of life outcomes · 2023Article
- Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints.Annals of neurology · 2023Review
- A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice.The Journal of clinical investigation · 2022Article
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
objectiveThe 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).
methodsInherited 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.
resultsThe 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.
interpretationThe CMTHI represents a valid and reliable outcome to assess patient-reported disease burden in CMT. Ann Neurol 2018;84:225-233.
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