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Muscle Nerve. 2016 Feb;53(2):183-90. doi: 10.1002/mus.24725. Epub 2015 Dec 29.

Myotonic dystrophy health index: Correlations with clinical tests and patient function.

Author information

1
The University of Rochester Medical Center, Department of Neurology, 601 Elmwood Avenue, Box 673, Rochester, New York, 14642, USA.
2
Psychometric Consultant, Chicago, Illinois, USA.
3
The University of Utah, Salt Lake City, Utah, USA.
4
The University of Rochester Medical Center, Department of Biostatistics and Computational Biology, Rochester, New York, USA.
5
The University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.
6
Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
7
David Geffen School of Medicine, UCLA Medical Center, Los Angeles, California, USA.
8
Greater Los Angeles VA HealthCare System, Los Angeles, California, USA.

Abstract

INTRODUCTION:

The Myotonic Dystrophy Health Index (MDHI) is a disease-specific patient-reported outcome measure. Here, we examine the associations between the MDHI and other measures of disease burden in a cohort of individuals with myotonic dystrophy type-1 (DM1).

METHODS:

We conducted a cross-sectional study of 70 patients with DM1. We examined the associations between MDHI total and subscale scores and scores from other clinical tests. Participants completed assessments of strength, myotonia, motor and respiratory function, ambulation, and body composition. Participants also provided blood samples, underwent physician evaluations, and completed other patient-reported outcome measures.

RESULTS:

MDHI total and subscale scores were strongly associated with muscle strength, myotonia, motor function, and other clinical measures.

CONCLUSIONS:

Patient-reported health status, as measured by the MDHI, is associated with alternative measures of clinical health. These results support the use of the MDHI as a valid tool to measure disease burden in DM1 patients.

KEYWORDS:

muscle disease; myotonic dystrophy Type-1; patient-relevant; patient-reported outcome measure; quality of life; therapeutic trial

PMID:
26044513
PMCID:
PMC4979973
DOI:
10.1002/mus.24725
[Indexed for MEDLINE]
Free PMC Article

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