NASHVILLE, Tennessee—There are a number of therapeutic choices for managing Duchenne muscular dystrophy (DMD), with each demonstrating benefits in clinical trials but also having potential risks and complications, Katherine Mathews, MD, said at a presentation at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.

However, there is no “one best choice” for every family since the circumstances of each one of them are different, said Dr. Mathews, professor of pediatrics-general neurology at the University of Iowa Carver College of Medicine in Iowa City.

“We’ve come a long way in neuromuscular therapeutics,” she said, recalling that she used to have only “a brief conversation” with her patients about their treatment options in 1995, when steroids were still not widely used for boys with DMD. Today, their efficacy has been proven, although their side effects are considerable. 


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According to Dr. Mathews, there are several practical considerations to keep in mind when choosing between prednisone and deflazacort, the 2 main corticosteroid treatments used to manage DMD, including cost, dosing regimen, and side effects. 

Interestingly, there does not seem to be a consensus on the relative effectiveness of different steroid regimens, according to Dr. Mathews.

“So what do we do when we are talking with families?” she said, stressing the importance of understanding what is the most important factor for families, such as side effects or cost. “Another thing that I think is really important to remind families is that they are not locked into one regimen,” Dr. Mathews said. “They can start with one and decide that now is the time to transition to another.”

There are also exon-skipping drugs available and evidence suggests they increase the amount of truncated dystrophin protein in muscles and slow disease progression. However, they are very expensive and require weekly intravenous infusions, which is a huge demand on a child and their family, according to Dr. Mathews. They are also associated with certain risks on top of the potential for renal toxicity.

“We encourage it but I certainly don’t insist that everybody do exon skipping,” Dr. Mathews said.

Challenges that doctors will face in the future as additional drugs are approved include choosing the best option for newly diagnosed patients and those who have been previously treated, using combination therapies, and deciding on the optimal timing for therapy administration. 

Reference

Mathews K. Therapeutic choices in Duchenne muscular dystrophy. Oral presentation at: 2022 Muscular Dystrophy Association Clinical & Scientific Conference; March 15, 2022; Nashville, TN.