New guideline: Corticosteroids recommended for duchenne muscular dystrophy

Corticosteroids can be beneficial in the treatment of Duchenne muscular dystrophy and can be offered as a treatment option, according to the American Academy of Neurology and the Child Neurology Society in a new practice guideline published in the January 11 issue of Neurology, the scientific journal of the American Academy of Neurology.

Duchenne muscular dystrophy is a genetic disorder linked to the X-chromosome. It is the most common form of muscular dystrophy in children and occurs when the protein dystrophin is missing. This causes a gradual breakdown of muscles and a decline in muscle strength. Duchenne muscular dystrophy mainly affects boys. An estimated one in 3,500 males worldwide has the disorder, and each year approximately 400 boys in the United States are born with it. Symptoms usually appear between ages two and five and include frequent falls, large calf muscles, and difficulty running, jumping, and getting up. There is no cure.

The guideline authors reviewed all available research for the use of corticosteroids in the treatment of Duchenne muscular dystrophy. Corticosteroids are man-made drugs that are similar to the body’s hormone cortisone. Two corticosteroids, prednisone and deflazacort, were found to slow the rate of muscle deterioration, and are recommended as potential treatments to minimize the effect of Duchenne muscular dystrophy.

Prednisone was found to help muscle strength and function and should be offered as a treatment option. Deflazacort, a drug similar to prednisone, is also recommended as a treatment option but is not available in the United States at this time. “Corticosteroids are the only effective drugs in providing improvements in children with Duchenne muscular dystrophy,” said lead author Richard T. Moxley III, MD, of the University of Rochester in Rochester, N.Y.

The guideline authors caution that an offer of treatment with corticosteroids should include a balanced discussion of potential benefits and risks. Possible side effects of corticosteroid therapy should be closely monitored by a physician. Side effects can include weight gain, cushingoid appearance (a rounded face), cataracts, short stature, acne, excessive hair growth, gastrointestinal symptoms, and behavioral changes. A nutrition plan and exercise program may prevent some of these side effects. “If the side effects outweigh the benefits, it is recommended to lower the dose of the drug,” said Moxley. “If the dose is decreased, the level of improvement in muscle strength and function may not be as great.”

Other treatments for Duchenne muscular dystrophy include physical therapy to reduce muscle tightening, orthopedic support devices, and corrective orthopedic surgery to improve the ability to function.

Complete guidelines, a summary for physicians, and a version for patients and their families will be available on January 11 at www.aan.com/professionals/practice/guideline/index.cfm.