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

R. T MOXLEY; S ASHWAL; S PANDYA; A CONNOLLY; J FLORENCE; K MATHEWS; L BAUMBACH; C MCDONALD; M SUSSMAN; C WADE

doi:10.1212/01.WNL.0000148485.00049.B7

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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

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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

R. T MOXLEY, S ASHWAL, S PANDYA, A CONNOLLY, J FLORENCE, K MATHEWS, L BAUMBACH, C MCDONALD, M SUSSMAN and C WADE

Neurology, Vol.64(1), pp.13-20

2005

DOI: 10.1212/01.WNL.0000148485.00049.B7

PMID: 15642897

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Abstract

Background: The Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society develop practice parameters as strategies for patient management based on analysis of evidence. Objective: To review available evidence on corticosteroid treatment of boys with Duchenne dystrophy. Methods: Relevant literature was reviewed, abstracted, and classified. Recommendations were based on a four-tiered scheme of evidence classification, and areas for future research are defined. Results: Seven class I studies and numerous less rigorous trials all demonstrated that corticosteroid treatment for 6 months with prednisone (0.75 or 1.5 mg/kg/day) increased muscle strength, performance, and pulmonary function and significantly slowed the progression of weakness. Two class I trials examined the effect of lower dosage of prednisone (0.30 and 0.35 mg/kg/day), demonstrated lesser but similar benefits, and showed a lower frequency of side effects (e.g., weight gain). The only significant side effects in all class I trials were weight gain and development of a cushingoid facial appearance. One longer-term trial of daily prednisone (0.3 to 0.7 mg/kg/day), a class III study, showed prolongation of functional ability and slower progression of weakness in patients during 3 years of treatment. One class IV, open trial of alternate-day prednisone (2 mg/kg for 2 months, then two-thirds dose every other day) extended ambulation by approximately 2 years in treated compared with untreated patients. Deflazacort, a corticosteroid similar in structure to prednisone, produced similar improvement in muscle strength and function with a similar side effect profile. Conclusions: Prednisone has been demonstrated to have a beneficial effect on muscle strength and function in boys with Duchenne dystrophy and should be offered (at a dose of 0.75 mg/kg/day) as treatment. If side effects require a decrease in prednisone, tapering to dosages as low as 0.3 mg/kg/day gives less robust but significant improvement. Deflazacort (0.9 mg/kg/day) can also be used for the treatment of Duchenne dystrophy in countries in which it is available. Benefits and side effects of corticosteroid therapy need to be monitored. The offer of treatment with corticosteroids should include a balanced discussion of potential risks.

Neurology

Biological and medical sciences

Nervous system as a whole

Medical sciences

Diseases of striated muscles. Neuromuscular diseases

Nervous system (semeiology, syndromes)

Details

Title: Subtitle: 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
Creators: R. T MOXLEY - Department of Neurology, University of Rochester School of Medicine and Dentistry, NY, United States
S ASHWAL - Division of Child Neurology, Department of Pediatrics, Loma Linda University School of Medicine, Sacramento, United States
S PANDYA - Department of Neurology, University of Rochester School of Medicine and Dentistry, NY, United States
A CONNOLLY - Department of Neurology, Washington University School of Medicine, St. Louis, MO, France
J FLORENCE - Department of Neurology, Washington University School of Medicine, St. Louis, MO, France
K MATHEWS - Departments of Pediatrics and Neurology, University of Iowa Hospitals, Iowa City, United States
L BAUMBACH - Institute for Molecular Genetics, Baylor College of Medicine, Houston, TX, United States
C MCDONALD - Department of Physical Medicine and Rehabilitation, University of California-Davis School of Medicine, Sacramento, United States
M SUSSMAN - Department of Orthopedic Surgery, Shriners Hospitals for Children, Portland, OR, United States
C WADE - Department of Zoology and Physiology, University of Wyoming, Laramie, United States
Resource Type: Journal article
Publication Details: Neurology, Vol.64(1), pp.13-20
DOI: 10.1212/01.WNL.0000148485.00049.B7
PMID: 15642897
NLM abbreviation: Neurology
ISSN: 0028-3878
eISSN: 1526-632X
Publisher: Lippincott Williams & Wilkins; Hagerstown, MD
Language: English
Date published: 2005
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984020629502771

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