Report Viewer

NINDS CDE Notice of Copyright
Myotonic Dystrophy Health Index (MDHI)
Availability
Please email the author for information about obtaining the instrument:
 
The Neuromuscular Institute of Quality-of-Life Studies and Outcome Measure Development
Licensing opportunities for the MDHI instrument are available through the Office of Technology Transfer (OTT) at the University of Rochester Office of Technology Transfer (Search for technology number 6-2189).
 
Please visit these websites for more information about the instrument:
Classification
NeuroRehab Supplemental - Highly Recommended
Recommendations for Use: Indicated for studies requiring a Myotonic Dystrophy-Specific Health-Related Quality of Life measure.
 
Supplemental-Highly Recommended: Myotonic Dystrophy (DM)
Highly recommended for use as a primary or secondary outcome measure during myotonic dystrophy clinical trials. Highly recommended as a marker of disease severity for patients with myotonic dystrophy.
Short Description of Instrument
The MDHI is a disease-specific patient reported outcome measure specifically created for myotonic dystrophy type-1 (DM1). It was designed to measure a patient's health state and to be used as a responsive outcome measure during clinical trials. It measures a patient's perception of their total health and 17 areas of sub-health. Subscales include mobility, upper extremity function, ability to do activities, fatigue, pain, gastrointestinal issues, vision, communication, sleep, emotional issues, cognitive impairment, social satisfaction, social performance, myotonia, breathing, swallowing, and hearing.
Comments/Special Instructions
The MDHI is designed for use as a primary or secondary outcome measure in DM clinical trials. The instrument measures total health of a DM patient as well as 17 additional areas that are relevant to DM subhealth.
Scoring
The total MDHI score and a score for each subscale is produced each time a patient completes the instrument. Scores range from 0 to 100 with 100 representing the most severe disease. Standard values are available for the MDHI total score and each subscale score based on gender, age, CTG repeat length, education level, and employment status.
 
Administration time is 15-20 minutes and does not require supervision by clinical staff.
Scoring and Psychometric Properties
Scoring: The total MDHI score and a score for each subscale is produced each time a patient completes the instrument. Scores range from 0 to 100 with 100 representing the most severe disease. Standard values are available for the MDHI total score and each subscale score based on gender, age, CTG repeat length, education level, and employment status.
 
Administration time is 15-20 minutes and does not require supervision by clinical staff.
 
Psychometric Properties: The MDHI was developed for use specifically for the DM1 population. It was validated using standard techniques, a large DM1 patient population and has been tested for validity and reliability. DM1 patients (n= 278) identified the most relevant questions for the MDHI. After factor analysis, patient interviews, and test-retest reliability assessments were done to refine and evaluate this instrument. Questions 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. Initial 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. In addition, this instrument is used in multiple clinical trials including the DM1 EU-study OPTIMISTIC (Okkersen et al., 2018).
Rationale/Justification
Strengths: The MDHI is a validated instrument capable of measuring clinically meaningful changes in patient health during clinical trials.
 
Weaknesses: It is a disease specific instrument.
References
Key References:
Heatwole C, Bode R, Johnson N, Dekdebrun J, Dilek N, Heatwole M, Hilbert JE, Luebbe E, Martens W, Mcdermott MP, Rothrock N, Thornton C, Vickrey BG, Victorson D, Moxley R 3rd. Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure. Muscle Nerve. 2014 Jun;49(6):906-14.
 
Heatwole C, Bode R, Johnson N, et al. Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1). Neurology 2012;79:348-57.
 
Additional Reference:
Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmüller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium. Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial. Lancet Neurol. 2018 Aug;17(8):671-680.
 
NeuroRehab-Specific Reference:
Ho AK, Horton MC, Landwehrmeyer GB, Burgunder JM, Tennant A; European Huntington's Disease Network. Meaningful and Measurable Health Domains in Huntington's Disease: Large-Scale Validation of the Huntington's Disease Health-Related Quality of Life Questionnaire Across Severity Stages. Value Health. 2019 Jun;22(6):712-720.
 
Document last updated October 2024