Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

Anne M. Connolly; Elizabeth C. Malkus; Jerry R. Mendell; Kevin M. Flanigan; J. Philip Miller; Jeanine R. Schierbecker; Catherine A. Siener; Paul T. Golumbek; Craig M. Zaidman; Craig M. Mcdonald; Linda Johnson; Alina Nicorici; Peter I. Karachunski; John W. Day; Jason M. Kelecic; Linda P. Lowes; Lindsay N. Alfano; Basil T. Darras; Peter B. Kang; Janet Quigley; Amy E. Pasternak; Julaine M. Florence; Pallavi Anand; Charlie O. Wulf; Erica Goude; Joline C. Dalton; Laurence Viollet-Callendret; Karen K. Buser; Elizabeth Shriber; Rebecca Parad; MDA DMD Clinical Research Network

doi:10.1002/mus.24346

Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

Anne M. Connolly, Elizabeth C. Malkus, Jerry R. Mendell, Kevin M. Flanigan, J. Philip Miller, Jeanine R. Schierbecker, Catherine A. Siener, Paul T. Golumbek, Craig M. Zaidman, Craig M. Mcdonald, Linda Johnson, Alina Nicorici, Peter I. Karachunski, John W. Day, Jason M. Kelecic, Linda P. Lowes, Lindsay N. Alfano, Basil T. Darras, Peter B. Kang, Janet QuigleyAmy E. Pasternak, Julaine M. Florence, Pallavi Anand, Charlie O. Wulf, Erica Goude, Joline C. Dalton, Laurence Viollet-Callendret, Karen K. Buser, Elizabeth Shriber, Rebecca Parad, MDA DMD Clinical Research Network

Neurology

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

63 Scopus citations

Abstract

Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

Original language	English (US)
Pages (from-to)	522-532
Number of pages	11
Journal	Muscle and Nerve
Volume	51
Issue number	4
DOIs	https://doi.org/10.1002/mus.24346
State	Published - Apr 1 2015

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Publisher Copyright:
© 2014 Wiley Periodicals, Inc..

Keywords

Corticosteroids
Duchenne muscular dystrophy
Non-ambulatory
Pulmonary function
Quality of life
Strength

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10.1002/mus.24346

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Connolly, A. M., Malkus, E. C., Mendell, J. R., Flanigan, K. M., Miller, J. P., Schierbecker, J. R., Siener, C. A., Golumbek, P. T., Zaidman, C. M., Mcdonald, C. M., Johnson, L., Nicorici, A., Karachunski, P. I., Day, J. W., Kelecic, J. M., Lowes, L. P., Alfano, L. N., Darras, B. T., Kang, P. B., ... MDA DMD Clinical Research Network (2015). Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy. Muscle and Nerve, 51(4), 522-532. https://doi.org/10.1002/mus.24346

Connolly, AM, Malkus, EC, Mendell, JR, Flanigan, KM, Miller, JP, Schierbecker, JR, Siener, CA, Golumbek, PT, Zaidman, CM, Mcdonald, CM, Johnson, L, Nicorici, A, Karachunski, PI, Day, JW, Kelecic, JM, Lowes, LP, Alfano, LN, Darras, BT, Kang, PB, Quigley, J, Pasternak, AE, Florence, JM, Anand, P, Wulf, CO, Goude, E, Dalton, JC, Viollet-Callendret, L, Buser, KK, Shriber, E, Parad, R & MDA DMD Clinical Research Network 2015, 'Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy', Muscle and Nerve, vol. 51, no. 4, pp. 522-532. https://doi.org/10.1002/mus.24346

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title = "Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy",

abstract = "Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.",

keywords = "Corticosteroids, Duchenne muscular dystrophy, Non-ambulatory, Pulmonary function, Quality of life, Strength",

author = "Connolly, {Anne M.} and Malkus, {Elizabeth C.} and Mendell, {Jerry R.} and Flanigan, {Kevin M.} and Miller, {J. Philip} and Schierbecker, {Jeanine R.} and Siener, {Catherine A.} and Golumbek, {Paul T.} and Zaidman, {Craig M.} and Mcdonald, {Craig M.} and Linda Johnson and Alina Nicorici and Karachunski, {Peter I.} and Day, {John W.} and Kelecic, {Jason M.} and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Darras, {Basil T.} and Kang, {Peter B.} and Janet Quigley and Pasternak, {Amy E.} and Florence, {Julaine M.} and Pallavi Anand and Wulf, {Charlie O.} and Erica Goude and Dalton, {Joline C.} and Laurence Viollet-Callendret and Buser, {Karen K.} and Elizabeth Shriber and Rebecca Parad and {MDA DMD Clinical Research Network}",

note = "Publisher Copyright: {\textcopyright} 2014 Wiley Periodicals, Inc..",

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doi = "10.1002/mus.24346",

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T1 - Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

AU - Connolly, Anne M.

AU - Malkus, Elizabeth C.

AU - Mendell, Jerry R.

AU - Flanigan, Kevin M.

AU - Miller, J. Philip

AU - Schierbecker, Jeanine R.

AU - Siener, Catherine A.

AU - Golumbek, Paul T.

AU - Zaidman, Craig M.

AU - Mcdonald, Craig M.

AU - Johnson, Linda

AU - Nicorici, Alina

AU - Karachunski, Peter I.

AU - Day, John W.

AU - Kelecic, Jason M.

AU - Lowes, Linda P.

AU - Alfano, Lindsay N.

AU - Darras, Basil T.

AU - Kang, Peter B.

AU - Quigley, Janet

AU - Pasternak, Amy E.

AU - Florence, Julaine M.

AU - Anand, Pallavi

AU - Wulf, Charlie O.

AU - Goude, Erica

AU - Dalton, Joline C.

AU - Viollet-Callendret, Laurence

AU - Buser, Karen K.

AU - Shriber, Elizabeth

AU - Parad, Rebecca

AU - MDA DMD Clinical Research Network

PY - 2015/4/1

Y1 - 2015/4/1

N2 - Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

AB - Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

KW - Corticosteroids

KW - Duchenne muscular dystrophy

KW - Non-ambulatory

KW - Pulmonary function

KW - Quality of life

KW - Strength

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Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

Abstract

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Keywords

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