New AAN Practice Guideline Update Backs Use of Corticosteroids for Duchenne Muscular Dystrophy.

A new practice guideline looks at the effectiveness and side effect profile of long-term use of corticosteroids for Duchenne muscular dystrophy. The guideline replaces an earlier version published in 2005. Nearly 10 years after the publication of a practice guideline on corticosteroids for Duchenne muscular dystrophy (DMD), the AAN has provided an updated review of the evidence on their effectiveness for delaying and lessening the effects of certain symptoms and enhancing quality of life and survival. The practice parameter replaces a practice guideline published in 2005, when there was little published research on the long-term use of corticosteroids for DMD.

“Research has suggested that there is wide variation in corticosteroid use to treat patients with Duchenne muscular dystrophy,” said Richard T. Moxley III, MD, FAAN, a coauthor of the new guideline and lead author of the previous one. He said some practitioners have been hesitant to prescribe corticosteroids long-term because they worry that “when you continue treatment, the boys are going to have all these side effects.”

The new guideline, published in the February 2 issue of Neurology, is based on a review of 63 articles from 2004-2014 on the use of prednisone or deflazacort for DMD, including a few related studies on bone health interventions. Deflazacort is currently not available on the US market, but Marathon Pharmaceuticals hopes to gain Food and Drug Administration approval to market the drug for DMD, Dr. Moxley said.

“It is timely to provide care providers, patients, and their families with updated published information about the effectiveness of corticosteroid treatment, the treatment regimens that have received careful assessment, the side effects during short-term and long-term treatment, and the future challenges and opportunities ahead for corticosteroid therapy in these patients,” Dr. Moxley, the Helen Aresty Fine and Irving Fine professor of neurology at the University of Rochester, told Neurology Today.

Dr. Moxley discussed some of the guideline's recommendations and others that may be coming along to enhance treatment for DMD patients.

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