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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