Heart Drug Inspra Improves Cardiac Function in Young Boys with DMD, Study Shows

Administration of Inspra (eplerenone) to young boys with Duchenne muscular dystrophy (DMD) improves their heart function and stabilizes it in older boys, a recent study shows.

The study “Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: results of a two-year open-label extension trial” was published in the Orphanet Journal of Rare Diseases.

While cardiomyopathy, a term that defines diseases that affect the heart muscle, is relatively rare in children, it is a leading cause of morbidity and mortality in boys with DMD.

In a previous report, the team of scientists at Ohio State University Ross Heart Hospital and Nationwide Children’s Hospital performed a 12-month double-blind randomized controlled trial (NCT01521546) testing the effects of Inspra, a drug used in the management of high blood pressure and chronic heart failure. They found that Inspra slowed the progressive decline in heart function in DMD patients when compared to a placebo control.

This time the team performed a follow-up study and evaluated “the safety and efficacy of longer-term eplerenone therapy in boys with DMD.”

They followed 11 patients (all male aged 7 to 25) who previously had been enrolled in the first 12-month study. Now, they were followed for 24 months, during which time patients received a daily dose of 25 mg of Inspra to treat preclinical DMD cardiomyopathy.

They measured a parameter defined as left ventricular systolic strain, a sensitive measure of cardiac function that predicts cardiac problems even before symptoms are evident.

“We saw significant improvement in left ventricular systolic function among the younger boys who were newly treated with eplerenone. The older patients who continued eplerenone therapy from the previous trial to this one remained stable,” Subha Raman, MD, a cardiologist and professor at Ohio State Wexner Medical Center and study lead author, said in a press release. “Recognizing that cardiopulmonary failure remains the leading cause of death in this disease, this tells us we should strongly consider early use of this medication in boys with DMD in order to gain the greatest cardiac benefit.”

Importantly, Inspra induced no adverse effects, such as high potassium levels.

“Eplerenone offers effective and safe cardioprotection for boys with DMD, particularly when started at a younger age. Eplerenone is a useful clinical therapeutic option, particularly if treatment is initiated earlier in life when cardiac damage is minimal,” researchers concluded.

“It’s important to remember that the heart is a muscle, too. If we’re to achieve long-term improvements in duration and quality of life for patients with DMD, we must strive to understand the associated heart muscle disease better. This trial is one small piece of that big puzzle,” added Linda Cripe, MD, a pediatric cardiologist and co-investigator at Nationwide Children’s.