Cheaper, Generic Drug Just as Effective in Preserving Heart Function in Duchenne Muscular Dystrophy

A recent clinical trial conducted found a cost-effective generic medication works just as well as a more expensive drug in preserving heart function in boys with Duchenne muscular dystrophy. Published in the Journal of the American Heart Association, the study compared two mineralocorticoid receptor antagonist drugs and found them to be equally effective in preserving cardiac contractility.

Duchenne muscular dystrophy (DMD) is a genetic disorder affecting skeletal and heart muscles. Most patients are males and survive into their 20s or 30s.

Researchers compared two mineralocorticoid receptor antagonists, spironolactone and eplerenone. Previously, the team showed the success of eplerenone in protecting the heart in boys with DMD. The primary objective of this study, the AIDMD (Aldosterone Inhibition in Duchenne Muscular Dystrophy) trial, was to see if the generic drug spironolactone would provide similar cardioprotection to boys with DMD.

To conduct the trial, the researchers enrolled 52 boys with DMD between the ages of 12 and 18 at seven academic medical centers, including The Ohio State University Wexner Medical Center, across the country. After baseline cardiac imaging, participants were randomly assigned to receive either eplerenone or spironolactone daily for one year.

At the end of 12 months, no statistical difference was demonstrated in cardiac function between that groups, suggesting that at least in the short term that spironolactone and eplerenone are equivalent in cardioprotection.

“ We cannont forget that the heart is a muscle too.  These results reinforce the need to continue to explore treatments that impact the heart in DMD ,” says Linda Cripe, MD, a pediatric cardiologist at The Heart Center at Nationwide Children’s and one of the study’s authors. “These findings support the potential benefit of early treatment with mineralocorticoid receptor antagonist therapy.”

Dr. Cripe and her colleagues say that the low incidence of adverse events, along with the ready availability of these medications at low cost compared with other DMD therapies, provide support for the wider and earlier adoption of mineralocorticoid receptor antagonists – especially spironolactone, which is more commonly available worldwide and less expensive than eplerenone.

“This trial provided us with valuable information that will impact cardiac care, even though there remains much to learn about cardiomyopathy in DMD,” says Dr. Cripe.”

Reference:

Subha V. Raman, Kan N. Hor, Wojciech Mazur, Andrea Cardona, Xin He, Nancy Halnon, Larry Markham, Jonathan H. Soslow, Michael D. Puchalski, Scott R. Auerbach, Uyen Truong, Suzanne Smart, Beth McCarthy, Ibrahim M. Saeed, Jeffrey M. Statland, John T. Kissel, Linda H. Cripe. Stabilization of Early Duchenne Cardiomyopathy With Aldosterone Inhibition: Results of the Multicenter AIDMD Trial. Journal of the American Heart Association, 2019; 8 (19) DOI: 10.1161/JAHA.119.013501