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

Chad Heatwole; Rita Bode; Nicholas E. Johnson; Jeanne Dekdebrun; Nuran Dilek; Katy Eichinger; James E. Hilbert; Eric Logigian; Elizabeth Luebbe; William Martens; Michael P. Mcdermott; Shree Pandya; Araya Puwanant; Nan Rothrock; Charles Thornton; Barbara G. Vickrey; David Victorson; Richard T. Moxley

doi:10.1002/mus.24725

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

Chad Heatwole, Rita Bode, Nicholas E. Johnson, Jeanne Dekdebrun, Nuran Dilek, Katy Eichinger, James E. Hilbert, Eric Logigian, Elizabeth Luebbe, William Martens, Michael P. Mcdermott, Shree Pandya, Araya Puwanant, Nan Rothrock, Charles Thornton, Barbara G. Vickrey, David Victorson, Richard T. Moxley

Research output: Contribution to journal › Article › peer-review

34 Scopus citations

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.

Original language	English
Pages (from-to)	183-190
Number of pages	8
Journal	Muscle and Nerve
Volume	53
Issue number	2
DOIs	https://doi.org/10.1002/mus.24725
State	Published - 1 Feb 2016
Externally published	Yes

Keywords

Muscle disease
Myotonic dystrophy Type-1
Patient-relevant
Patient-reported outcome measure
Quality of life
Therapeutic trial

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Heatwole, C., Bode, R., Johnson, N. E., Dekdebrun, J., Dilek, N., Eichinger, K., Hilbert, J. E., Logigian, E., Luebbe, E., Martens, W., Mcdermott, M. P., Pandya, S., Puwanant, A., Rothrock, N., Thornton, C., Vickrey, B. G., Victorson, D., & Moxley, R. T. (2016). Myotonic dystrophy health index: Correlations with clinical tests and patient function. Muscle and Nerve, 53(2), 183-190. https://doi.org/10.1002/mus.24725

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title = "Myotonic dystrophy health index: Correlations with clinical tests and patient function",

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",

author = "Chad Heatwole and Rita Bode and Johnson, {Nicholas E.} and Jeanne Dekdebrun and Nuran Dilek and Katy Eichinger and Hilbert, {James E.} and Eric Logigian and Elizabeth Luebbe and William Martens and Mcdermott, {Michael P.} and Shree Pandya and Araya Puwanant and Nan Rothrock and Charles Thornton and Vickrey, {Barbara G.} and David Victorson and Moxley, {Richard T.}",

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year = "2016",

month = feb,

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doi = "10.1002/mus.24725",

language = "English",

volume = "53",

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Heatwole, C, Bode, R, Johnson, NE, Dekdebrun, J, Dilek, N, Eichinger, K, Hilbert, JE, Logigian, E, Luebbe, E, Martens, W, Mcdermott, MP, Pandya, S, Puwanant, A, Rothrock, N, Thornton, C, Vickrey, BG, Victorson, D & Moxley, RT 2016, 'Myotonic dystrophy health index: Correlations with clinical tests and patient function', Muscle and Nerve, vol. 53, no. 2, pp. 183-190. https://doi.org/10.1002/mus.24725

TY - JOUR

T1 - Myotonic dystrophy health index

T2 - Correlations with clinical tests and patient function

AU - Heatwole, Chad

AU - Bode, Rita

AU - Johnson, Nicholas E.

AU - Dekdebrun, Jeanne

AU - Dilek, Nuran

AU - Eichinger, Katy

AU - Hilbert, James E.

AU - Logigian, Eric

AU - Luebbe, Elizabeth

AU - Martens, William

AU - Mcdermott, Michael P.

AU - Pandya, Shree

AU - Puwanant, Araya

AU - Rothrock, Nan

AU - Thornton, Charles

AU - Vickrey, Barbara G.

AU - Victorson, David

AU - Moxley, Richard T.

PY - 2016/2/1

Y1 - 2016/2/1

N2 - 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.

AB - 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.

KW - Muscle disease

KW - Myotonic dystrophy Type-1

KW - Patient-relevant

KW - Patient-reported outcome measure

KW - Quality of life

KW - Therapeutic trial

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U2 - 10.1002/mus.24725

DO - 10.1002/mus.24725

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C2 - 26044513

AN - SCOPUS:84956585749

SN - 0148-639X

VL - 53

SP - 183

EP - 190

JO - Muscle and Nerve

JF - Muscle and Nerve

IS - 2

ER -

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

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Keywords

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