ArticleMuscle & nerve2014
Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure.
Article in Muscle & nerve, 2014. 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
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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.
- Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial.Neurology · 2021Trial
- 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
- Exploring the impact of myotonia on daily functioning in myotonic dystrophy: a patient-reported survey.BMC neurology · 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
- Observational
- RNA mis-splicing in children with congenital myotonic dystrophy is associated with physical function.Annals of clinical and translational neurology · 2024Article
- Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.Neurology. Clinical practice · 2024Article
- Friedreich's Ataxia-Health Index: Development and Validation of a Novel Disease-Specific Patient-Reported Outcome Measure.Neurology. Clinical practice · 2023Article
- Brief assessment of cognitive function in myotonic dystrophy: Multicenter longitudinal study using computer-assisted evaluation.Muscle & nerve · 2022Observational
- Blood Transcriptome Profiling Links Immunity to Disease Severity in Myotonic Dystrophy Type 1 (DM1).International journal of molecular sciences · 2022Article
- Quality of life and subjective symptom impact in Japanese patients with myotonic dystrophy type 1.BMC neurology · 2022Article
- White matter microstructure relates to motor outcomes in myotonic dystrophy type 1 independently of disease duration and genetic burden.Scientific reports · 2021Article
- Central Nervous System Involvement as Outcome Measure for Clinical Trials Efficacy in Myotonic Dystrophy Type 1.Frontiers in neurology · 2020Review
- Towards Central Nervous System Involvement in Adults with Hereditary Myopathies.Journal of neuromuscular diseases · 2020Review
- Endurance exercise leads to beneficial molecular and physiological effects in a mouse model of myotonic dystrophy type 1.Muscle & nerve · 2019Article
- Analysis of the functional capacity outcome measures for myotonic dystrophy.Annals of clinical and translational neurology · 2019Article
- The myotonic dystrophy experience: a North American cross-sectional study.Muscle & nerve · 2019Article
- The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome.Annals of neurology · 2018Article
- Patient-identified impact of symptoms in spinal and bulbar muscular atrophy.Muscle & nerve · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
Abstract
introductionIn preparation for clinical trials we examine the validity, reliability, and patient understanding of the Myotonic Dystrophy Health Index (MDHI).
methodsInitially we partnered with 278 myotonic dystrophy type-1 (DM1) patients and identified the most relevant questions for the MDHI. Next, we used factor analysis, patient interviews, and test-retest reliability assessments to refine and evaluate the instrument. Lastly, we determined the capability of the MDHI to differentiate between known groups of DM1 participants.
resultsQuestions in the final MDHI represent 17 areas of DM1 health. The internal consistency was acceptable in all subscales. The MDHI had a high test-retest reliability (ICC = 0.95) and differentiated between DM1 patient groups with different disease severities.
conclusionsInitial evaluation of the MDHI provides evidence that it is valid and reliable as an outcome measure for assessing patient-reported health. These results suggest that important aspects of DM1 health may be measured effectively using the MDHI.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.