Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up

Anne M. Connolly; Julaine M. Florence; Craig M. Zaidman; Paul T. Golumbek; Jerry R. Mendell; Kevin M. Flanigan; Peter I. Karachunski; John W. Day; Craig M. McDonald; Basil T. Darras; Peter B. Kang; Catherine A. Siener; Rebecca K. Gadeken; Pallavi Anand; Jeanine R. Schierbecker; Elizabeth C. Malkus; Linda P. Lowes; Lindsay N. Alfano; Linda Johnson; Alina Nicorici; Jason M. Kelecic; Janet Quigley; Amy E. Pasternak; J. Philip Miller

doi:10.1002/mus.25089

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up

Anne M. Connolly, Julaine M. Florence, Craig M. Zaidman, Paul T. Golumbek, Jerry R. Mendell, Kevin M. Flanigan, Peter I. Karachunski, John W. Day, Craig M. McDonald, Basil T. Darras, Peter B. Kang, Catherine A. Siener, Rebecca K. Gadeken, Pallavi Anand, Jeanine R. Schierbecker, Elizabeth C. Malkus, Linda P. Lowes, Lindsay N. Alfano, Linda Johnson, Alina NicoriciJason M. Kelecic, Janet Quigley, Amy E. Pasternak, J. Philip Miller

Neurology

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

30 Scopus citations

Abstract

Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016.

Original language	English (US)
Pages (from-to)	681-689
Number of pages	9
Journal	Muscle and Nerve
Volume	54
Issue number	4
DOIs	https://doi.org/10.1002/mus.25089
State	Published - Oct 1 2016

Bibliographical note

Publisher Copyright:
© 2016 Wiley Periodicals, Inc.

Keywords

Brooke Scale
Duchenne muscular dystrophy
Egen Klassifikation Scale
clinical trial
force vital capacity
non-ambulatory
outcomes

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10.1002/mus.25089

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Connolly, A. M., Florence, J. M., Zaidman, C. M., Golumbek, P. T., Mendell, J. R., Flanigan, K. M., Karachunski, P. I., Day, J. W., McDonald, C. M., Darras, B. T., Kang, P. B., Siener, C. A., Gadeken, R. K., Anand, P., Schierbecker, J. R., Malkus, E. C., Lowes, L. P., Alfano, L. N., Johnson, L., ... Miller, J. P. (2016). Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up. Muscle and Nerve, 54(4), 681-689. https://doi.org/10.1002/mus.25089

Connolly, AM, Florence, JM, Zaidman, CM, Golumbek, PT, Mendell, JR, Flanigan, KM, Karachunski, PI, Day, JW, McDonald, CM, Darras, BT, Kang, PB, Siener, CA, Gadeken, RK, Anand, P, Schierbecker, JR, Malkus, EC, Lowes, LP, Alfano, LN, Johnson, L, Nicorici, A, Kelecic, JM, Quigley, J, Pasternak, AE & Miller, JP 2016, 'Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up', Muscle and Nerve, vol. 54, no. 4, pp. 681-689. https://doi.org/10.1002/mus.25089

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title = "Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up",

abstract = "Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016.",

keywords = "Brooke Scale, Duchenne muscular dystrophy, Egen Klassifikation Scale, clinical trial, force vital capacity, non-ambulatory, outcomes",

author = "Connolly, {Anne M.} and Florence, {Julaine M.} and Zaidman, {Craig M.} and Golumbek, {Paul T.} and Mendell, {Jerry R.} and Flanigan, {Kevin M.} and Karachunski, {Peter I.} and Day, {John W.} and McDonald, {Craig M.} and Darras, {Basil T.} and Kang, {Peter B.} and Siener, {Catherine A.} and Gadeken, {Rebecca K.} and Pallavi Anand and Schierbecker, {Jeanine R.} and Malkus, {Elizabeth C.} and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Linda Johnson and Alina Nicorici and Kelecic, {Jason M.} and Janet Quigley and Pasternak, {Amy E.} and Miller, {J. Philip}",

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doi = "10.1002/mus.25089",

language = "English (US)",

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T1 - Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy

T2 - MDA–DMD network follow-up

AU - Connolly, Anne M.

AU - Florence, Julaine M.

AU - Zaidman, Craig M.

AU - Golumbek, Paul T.

AU - Mendell, Jerry R.

AU - Flanigan, Kevin M.

AU - Karachunski, Peter I.

AU - Day, John W.

AU - McDonald, Craig M.

AU - Darras, Basil T.

AU - Kang, Peter B.

AU - Siener, Catherine A.

AU - Gadeken, Rebecca K.

AU - Anand, Pallavi

AU - Schierbecker, Jeanine R.

AU - Malkus, Elizabeth C.

AU - Lowes, Linda P.

AU - Alfano, Lindsay N.

AU - Johnson, Linda

AU - Nicorici, Alina

AU - Kelecic, Jason M.

AU - Quigley, Janet

AU - Pasternak, Amy E.

AU - Miller, J. Philip

PY - 2016/10/1

Y1 - 2016/10/1

N2 - Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016.

AB - Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016.

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KW - Duchenne muscular dystrophy

KW - Egen Klassifikation Scale

KW - clinical trial

KW - force vital capacity

KW - non-ambulatory

KW - outcomes

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ER -

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up

Abstract

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Keywords

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