This Article  • References  • Full text PDF  • Reply to article  • Send to a friend  • Save in My Folder  • Save to citation manager  • Permissions  Citing Articles  • Citation map  • Citing articles on HighWire  • Contact me when this article is cited  Related Content  • Similar articles in this journal  Social Bookmarking   What's this?

A Randomized, Controlled Trial Defining the Time Course and Dose Response

Robert C. Griggs, MD; Richard T. Moxley III, MD; Jerry R. Mendell, MD; Gerald M. Fenichel, MD; Michael H. Brooke, MD; Alan Pestronk, MD; J. Philip Miller; the Clinical Investigation of Duchenne Dystrophy Group

Arch Neurol. 1991;48(4):383-388.

Abstract
• A randomized, controlled trial of daily prednisone was conducted in 99 boys (aged 5 to 15 years) with Duchenne dystrophy to define the time course of improvement and the dose response to treatment. Prednisone at 0.3 mg/kg (n=33), prednisone at 0.75 mg/kg (n = 34), and placebo (n = 32) were administered for 6 months. Patients were examined using manual muscle and myometry testing, timed functional testing, pulmonary function testing, and laboratory measurements at 10 days, 1 month, 2 months, 3 months, and 6 months of treatment. Boys treated with prednisone had stronger average muscle strength scores, than did boys treated with placebo as early as 10 days after starting therapy. At the 3-month visit, the boys in the group given 0.75 mg/kg of prednisone were significantly stronger than those in the group given 0.3 mg/kg of prednisone, indicating a dose response. At 6 months, significant side effects occurred in the group treated with 0.75 mg/kg of prednisone, including weight gain, cushingoid appearance, and excessive hair growth. Only weight gain was observed in the group taking prednisone at a dose of 0.3 mg/kg. Importantly, no side effects were evident at 10 days or 1 month of treatment, despite improvement in muscle strength and function. We conclude that prednisone produces a rapid increase in muscle strength in patients with Duchenne dystrophy and that this improvement is maximal at a prednisone dosage of 0.75 mg/kg or less.

Author Affiliations
From the Department of Neurology, University of Rochester (NY) (Drs Griggs and Moxley); Department of Neurology, Vanderbilt University, Nashville, Tenn (Dr Fenichel); Department of Neurology and Neurosurgery, Washington University School of Medicine (Dr Pestronk), and Division of Biostatistics, Washington University (Mr Miller). St Louis, Mo; Department of Neurology, The Ohio State University College of Medicine, Columbus (Dr Mendell); and Division of Neurology, Walter C. McKenzie Center for Health Sciences, Edmonton, Alberta (Dr Brooke).

Footnotes
Accepted for publication November 27, 1990.

Reprint requests to Department of Neurology, University of Rochester, Box 673, 601 Elmwood Ave, Rochester, NY 14642 (Dr Griggs).

CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    Twitter     What's this?

THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES

Mechanisms of exercise limitation and pulmonary rehabilitation for patients with neuromuscular disease
Aboussouan
Chronic Respiratory Disease 2009;6:231-249.
ABSTRACT  

Albuterol increases lean body mass in ambulatory boys with Duchenne or Becker muscular dystrophy
Skura et al.
Neurology 2008;70:137-143.
ABSTRACT | FULL TEXT  

Effect of Long-term Steroids on Cough Efficiency and Respiratory Muscle Strength in Patients With Duchenne Muscular Dystrophy
Daftary et al.
Pediatrics 2007;119:e320-e324.
ABSTRACT | FULL TEXT  

The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy
Bonifati et al.
J. Neurol. Neurosurg. Psychiatry 2006;77:1177-1179.
ABSTRACT | FULL TEXT  

Expression and Regulation of CC Class Chemokines in the Dystrophic (mdx) Diaphragm
Demoule et al.
Am. J. Respir. Cell Mol. Bio. 2005;33:178-185.
ABSTRACT | FULL TEXT  

Practice Parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society
Moxley et al.
Neurology 2005;64:13-20.
ABSTRACT | FULL TEXT  

Intermittent Prednisone Therapy in Duchenne Muscular Dystrophy: A Randomized Controlled Trial
Beenakker et al.
Arch Neurol 2005;62:128-132.
ABSTRACT | FULL TEXT  

Respiratory Care of the Patient with Duchenne Muscular Dystrophy: ATS Consensus Statement
Am. J. Respir. Crit. Care Med. 2004;170:456-465.
FULL TEXT  

Pilot trial of albuterol in Duchenne and Becker muscular dystrophy
Fowler et al.
Neurology 2004;62:1006-1008.
ABSTRACT | FULL TEXT  

Steroid Treatment and the Development of Scoliosis in Males with Duchenne Muscular Dystrophy
Alman et al.
JBJS 2004;86:519-524.
ABSTRACT | FULL TEXT  

Corticosteroids in Duchenne Muscular Dystrophy: A Reappraisal
Wong and Christopher
J Child Neurol 2002;17:183-190.
ABSTRACT  

Prednisone Therapy in Becker's Muscular Dystrophy
Johnsen and Clinic
J Child Neurol 2001;16:870-871.
ABSTRACT  

A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy
Fenichel et al.
Neurology 2001;56:1075-1079.
ABSTRACT | FULL TEXT