Trial Suggests Early Drug Treatment
Prolongs Heart Function in DMD

In a study of 51 boys with Duchenne muscular dystrophy (DMD), a French research group found that early treatment with 2 to 4 milligrams a day of the drug perindopril prevented the development of serious cardiac dysfunction when patients began taking it while their heart function was still normal. (Cardiac function was evaluated by measuring “left ventricular ejection fraction,” which is how much blood the left lower chamber of the heart ejects with each contraction.)

Denis Duboc at Cochin Hospital in Paris, with colleagues at 10 institutions, randomly assigned the study participants to receive perindopril, which belongs to a class of drugs known as ACE (angiotensin converting enzyme) inhibitors, or a placebo (inert substance). All participants were boys with DMD between 9.5 and 13 years old, and all had normal cardiac function at the start of the study.

After three years, there were no significant differences between the perindopril group and the placebo group. At that time, perindopril was given to all participants.

After two additional years, the researchers compared the boys who had received the drug for all five years with those who started the drug three years into the study. One boy who had taken perindopril for all five years showed significant cardiac function loss, but eight participants who had taken it for only two years showed this type of loss.

The investigators concluded that “studies of preventive treatment with perindopril at a younger age are warranted.”

Elizabeth McNally, a cardiologist and MDA research grantee at the University of Chicago, commented on the findings, which are in the March 15 issue of the Journal of the American College of Cardiology. “The key finding is that early treatment was associated with fewer patients developing a more severe decline in heart function (one out of 24 versus eight out of 24),” she said.

She noted that all medications in the same class as perindopril will likely have the same effect. “ACE inhibition is the mainstay of therapy for cardiomyopathy [cardiac muscle dysfunction] in general, so it’s very reasonable to use it early in DMD to see if it slows the progression of developing cardiomyopathy.”

McNally added that there are additional medications, such as a class called beta blockers, that are also very effective in cardiomyopathy. “Those medications should be similarly tested,” she said.