Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

D. M. Escolar; L. P. Hache; P. R. Clemens; A. Cnaan; C. M. McDonald; V. Viswanathan; A. J. Kornberg; T. E. Bertorini; Y. Nevo; T. Lotze; A. Pestronk; M. M. Ryan; E. Monasterio; J. W. Day; A. Zimmerman; A. Arrieta; E. Henricson; J. Mayhew; J. Florence; F. Hu; A. M. Connolly

doi:10.1212/WNL.0b013e318227b164

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

D. M. Escolar, L. P. Hache, P. R. Clemens, A. Cnaan, C. M. McDonald, V. Viswanathan, A. J. Kornberg, T. E. Bertorini, Y. Nevo, T. Lotze, A. Pestronk, M. M. Ryan, E. Monasterio, J. W. Day, A. Zimmerman, A. Arrieta, E. Henricson, J. Mayhew, J. Florence, F. HuA. M. Connolly

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

103 Scopus citations

Abstract

Objective: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). Methods: A total of 64 boys withDMDwho were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months. Results: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone. Conclusions: Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. Classification of evidence: This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.

Original language	English (US)
Pages (from-to)	444-452
Number of pages	9
Journal	Neurology
Volume	77
Issue number	5
DOIs	https://doi.org/10.1212/WNL.0b013e318227b164
State	Published - Aug 2 2011
Externally published	Yes

ASJC Scopus subject areas

Clinical Neurology

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Escolar, D. M., Hache, L. P., Clemens, P. R., Cnaan, A., McDonald, C. M., Viswanathan, V., Kornberg, A. J., Bertorini, T. E., Nevo, Y., Lotze, T., Pestronk, A., Ryan, M. M., Monasterio, E., Day, J. W., Zimmerman, A., Arrieta, A., Henricson, E., Mayhew, J., Florence, J., ... Connolly, A. M. (2011). Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology, 77(5), 444-452. https://doi.org/10.1212/WNL.0b013e318227b164

Escolar, DM, Hache, LP, Clemens, PR, Cnaan, A, McDonald, CM, Viswanathan, V, Kornberg, AJ, Bertorini, TE, Nevo, Y, Lotze, T, Pestronk, A, Ryan, MM, Monasterio, E, Day, JW, Zimmerman, A, Arrieta, A, Henricson, E, Mayhew, J, Florence, J, Hu, F & Connolly, AM 2011, 'Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy', Neurology, vol. 77, no. 5, pp. 444-452. https://doi.org/10.1212/WNL.0b013e318227b164

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title = "Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy",

abstract = "Objective: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). Methods: A total of 64 boys withDMDwho were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months. Results: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone. Conclusions: Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. Classification of evidence: This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.",

author = "Escolar, {D. M.} and Hache, {L. P.} and Clemens, {P. R.} and A. Cnaan and McDonald, {C. M.} and V. Viswanathan and Kornberg, {A. J.} and Bertorini, {T. E.} and Y. Nevo and T. Lotze and A. Pestronk and Ryan, {M. M.} and E. Monasterio and Day, {J. W.} and A. Zimmerman and A. Arrieta and E. Henricson and J. Mayhew and J. Florence and F. Hu and Connolly, {A. M.}",

note = "Funding Information: Study funding: Supported by the Muscular Dystrophy Association (MDA), General Clinical Research Center (GCRC) 5M01 RR020359 , and the NIH ( K23 RR16281–01 ). ",

year = "2011",

month = aug,

day = "2",

doi = "10.1212/WNL.0b013e318227b164",

language = "English (US)",

volume = "77",

pages = "444--452",

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TY - JOUR

T1 - Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

AU - Escolar, D. M.

AU - Hache, L. P.

AU - Clemens, P. R.

AU - Cnaan, A.

AU - McDonald, C. M.

AU - Viswanathan, V.

AU - Kornberg, A. J.

AU - Bertorini, T. E.

AU - Nevo, Y.

AU - Lotze, T.

AU - Pestronk, A.

AU - Ryan, M. M.

AU - Monasterio, E.

AU - Day, J. W.

AU - Zimmerman, A.

AU - Arrieta, A.

AU - Henricson, E.

AU - Mayhew, J.

AU - Florence, J.

AU - Hu, F.

AU - Connolly, A. M.

N1 - Funding Information: Study funding: Supported by the Muscular Dystrophy Association (MDA), General Clinical Research Center (GCRC) 5M01 RR020359 , and the NIH ( K23 RR16281–01 ).

PY - 2011/8/2

Y1 - 2011/8/2

N2 - Objective: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). Methods: A total of 64 boys withDMDwho were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months. Results: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone. Conclusions: Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. Classification of evidence: This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.

AB - Objective: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). Methods: A total of 64 boys withDMDwho were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months. Results: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone. Conclusions: Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. Classification of evidence: This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.

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SN - 0028-3878

VL - 77

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JO - Neurology

JF - Neurology

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ER -

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

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