Myotonic Dystrophy Health Index: Initial evaluation of a disease-specific outcome measure

Chad Heatwole, Rita Bode, Nicholas Johnson, Jeanne Dekdebrun, Nuran Dilek, Mark Heatwole, James E. Hilbert, Elizabeth Luebbe, William Martens, Michael P. Mcdermott, Nan Rothrock, Charles Thornton, Barbara G. Vickrey, David Victorson, Richard Moxley

Research output: Contribution to journalArticlepeer-review

46 Scopus citations


Introduction: In preparation for clinical trials we examine the validity, reliability, and patient understanding of the Myotonic Dystrophy Health Index (MDHI). Methods: Initially 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. Results: 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. Conclusions: 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.

Original languageEnglish
Pages (from-to)906-914
Number of pages9
JournalMuscle and Nerve
Issue number6
StatePublished - Jun 2014
Externally publishedYes


  • Muscle disease
  • Myotonic dystrophy type-1
  • Patient-relevant
  • Patient-reported outcome measure
  • Therapeutic trial, quality of life


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