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

doi:10.1002/mus.24097

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 journal › Article › peer-review

53 Scopus citations

Abstract

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 language	English
Pages (from-to)	906-914
Number of pages	9
Journal	Muscle and Nerve
Volume	49
Issue number	6
DOIs	https://doi.org/10.1002/mus.24097
State	Published - Jun 2014
Externally published	Yes

Keywords

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

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10.1002/mus.24097

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Heatwole, C., Bode, R., Johnson, N., Dekdebrun, J., Dilek, N., Heatwole, M., Hilbert, J. E., Luebbe, E., Martens, W., Mcdermott, M. P., Rothrock, N., Thornton, C., Vickrey, B. G., Victorson, D., & Moxley, R. (2014). Myotonic Dystrophy Health Index: Initial evaluation of a disease-specific outcome measure. Muscle and Nerve, 49(6), 906-914. https://doi.org/10.1002/mus.24097

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title = "Myotonic Dystrophy Health Index: Initial evaluation of a disease-specific outcome measure",

abstract = "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.",

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

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

year = "2014",

month = jun,

doi = "10.1002/mus.24097",

language = "English",

volume = "49",

pages = "906--914",

journal = "Muscle and Nerve",

issn = "0148-639X",

publisher = "John Wiley \& Sons Inc.",

number = "6",

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TY - JOUR

T1 - Myotonic Dystrophy Health Index

T2 - Initial evaluation of a disease-specific outcome measure

AU - Heatwole, Chad

AU - Bode, Rita

AU - Johnson, Nicholas

AU - Dekdebrun, Jeanne

AU - Dilek, Nuran

AU - Heatwole, Mark

AU - Hilbert, James E.

AU - Luebbe, Elizabeth

AU - Martens, William

AU - Mcdermott, Michael P.

AU - Rothrock, Nan

AU - Thornton, Charles

AU - Vickrey, Barbara G.

AU - Victorson, David

AU - Moxley, Richard

PY - 2014/6

Y1 - 2014/6

N2 - 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.

AB - 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.

KW - Muscle disease

KW - Myotonic dystrophy type-1

KW - Patient-relevant

KW - Patient-reported outcome measure

KW - Therapeutic trial, quality of life

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AN - SCOPUS:84900507864

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EP - 914

JO - Muscle and Nerve

JF - Muscle and Nerve

IS - 6

ER -

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

Abstract

Keywords

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