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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 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
Footnotes
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Study funding: Supported by the Muscular Dystrophy Association (MDA), General Clinical Research Center (GCRC) 5M01 RR020359, and the NIH (K23 RR16281–01).
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The authors take full responsibility for the contents of this article, which do not represent the views of the Department of Veterans Affairs or the United States Government.
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Editorial, page 416
- Received March 25, 2010.
- Accepted February 10, 2011.
- Copyright © 2011 by AAN Enterprises, Inc.
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