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

D. M. Escolar, L. P. Hache, P. R. Clemens, A. Cnaan, Craig 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, Erik K Henricson, J. Mayhew, J. Florence, F. Hu & 1 others A. M. Connolly

Research output: Contribution to journalArticle

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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 languageEnglish (US)
Pages (from-to)444-452
Number of pages9
JournalNeurology
Volume77
Issue number5
DOIs
StatePublished - Aug 2 2011

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Duchenne Muscular Dystrophy
Prednisone
Muscles
Safety
Muscle Strength
Elbow
Double-Blind Method
Bone Density
Cataract
Leg
Appointments and Schedules
Body Mass Index
Arm
Blood Pressure
Weights and Measures
Research

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Escolar, D. M., Hache, L. P., Clemens, P. R., Cnaan, A., McDonald, C. M., Viswanathan, V., ... 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

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. / Escolar, D. M.; Hache, L. P.; Clemens, P. R.; Cnaan, A.; McDonald, Craig 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, Erik K; Mayhew, J.; Florence, J.; Hu, F.; Connolly, A. M.

In: Neurology, Vol. 77, No. 5, 02.08.2011, p. 444-452.

Research output: Contribution to journalArticle

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, EK, 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
Escolar, D. M. ; Hache, L. P. ; Clemens, P. R. ; Cnaan, A. ; McDonald, Craig 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, Erik K ; Mayhew, J. ; Florence, J. ; Hu, F. ; Connolly, A. M. / Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. In: Neurology. 2011 ; Vol. 77, No. 5. pp. 444-452.
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AU - Hache, L. P.

AU - Clemens, P. R.

AU - Cnaan, A.

AU - McDonald, Craig 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, Erik K

AU - Mayhew, J.

AU - Florence, J.

AU - Hu, F.

AU - Connolly, A. M.

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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.

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