Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.
Escolar, D.M. MD; Hache, L.P. MS; Clemens, P.R. MD; Cnaan, A. PhD; McDonald, C.M. MD; Viswanathan, V. MD; Kornberg, A.J. MD; Bertorini, T.E. MD; Nevo, Y. MD; Lotze, T. MD; Pestronk, A. MD; Ryan, M.M. MD; Monasterio, E. MD; Day, J.W. MD; Zimmerman, A. MS; Arrieta, A. MS; Henricson, E. MPH; Mayhew, J. Pt; Florence, J. Dpt; Hu, F. MS; Connolly, A.M. MD
77(5):444-452, August 2, 2011.
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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 with DMD who 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.
GLOSSARY: ANOVA: analysis of variance
BMI: body mass index
CBCL: Child Behavior Check List
CINRG: Cooperative International Neuromuscular Research Group
DEXA: dual-energy x-ray absorptiometry
DMD: Duchenne muscular dystrophy
FEV1: forced expiratory volume in 1 second
FVC: forced vital capacity
MIP: maximum inspiratory pressure
MMT: manual muscle testing
MVV: maximal voluntary ventilation
NCI: National Cancer Institute
PFT: pulmonary function test
QMT: quantitative muscle testing
(C)2011 American Academy of Neurology