Study Supports Early ACE Inhibitor Treatment in DMD

A 10-year French study of 57 boys with Duchenne muscular dystrophy (DMD) who were ages 9 to 13 at study entry found that early treatment with the angiotensin-converting enzyme inhibitor perindopril is associated with a survival benefit. ACE inhibitors reduce stress on the heart and are part of standard therapy for cardiac muscle dysfunction (cardiomyopathy) from any cause.

Denis Duboc at Cochin Hospital in Paris and colleagues, who published their findings in the September issue of American Heart Journal, randomly assigned 28 DMD patients with normal heart function to take 2 to 4 milligrams of perindopril per day; and another 29 patients, also with normal heart function, to take a placebo (inert substance).

After three years, there were no differences in cardiac function or survival in the two groups. Perindopril was then given to all study participants for up to 10 years.

At 10 years, 26 of 28 (93 percent) of the patients in the early perindopril group were alive, compared with only 19 of 29 (66 percent) who received a placebo for the first three years.

In the United States, the American Academy of Pediatrics recommended in 2005 that doctors consider using ACE inhibitors and/or another class of cardiac drugs, beta blockers, in children with DMD as soon as signs of cardiac dysfunction appear. However, use of ACE inhibitors or other medications before signs of cardiomyopathy are present is not yet standard treatment in French or U.S. clinics.