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Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure
Chad Heatwole1, Rita Bode, Nicholas Johnson
1The University of Rochester Medical Center, Department of Neurology, 601 Elmwood Avenue, Box 673, Rochester, New York, 14642, USA.
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.
