A study led by a cardiologist at The Ohio State University Wexner Medical Center found that administering medication for heart failure as early as possible can help slow progressive decline in cardiac function, commonly observed in young boys diagnosed with Duchenne muscular dystrophy (DMD). Lead author Dr. Subha Raman said, “We believe this research offers evidence that supports the early use of these readily available medications.”
Together with a team of experts on this rare and fatal disease, Dr. Raman carried out a clinical trial testing a combined treatment of eplerenone and either an ACE inhibitor or an angiotensin receptor blocker (ARB) in decreasing the rate of cardiac failure in boys with DMD. Her co-researcher and pediatric cardiologist, Dr. Linda Cripe from the Nationwide Children’s Hospital believes the results of this study could potentially change DMD treatment standards.
This clinical trial follows encouraging evidence from previous tests on animal DMD models, demonstrating the efficacy of combined pharmacologic agents in reducing muscle damage. Raman’s randomized, double-blind study treated 42 DMD patients that showed cardiac muscle damage on MRI scans. Each patient would receive either one daily dose of 25 mg eplerenone or a placebo for a year, while receiving adjuvant treatment with a physician-prescribed ACE inhibitor or ARB. The study completed enrollment along with necessary follow up appointments between March 2012 and July 2014.
The participants underwent cardiac MRIs before treatment was administered, again at 6 months into treatment, and a last scan at 12 months. The researchers were then able to observe a significant reduction in the decline in left ventricular function among patients who received eplerenone. Dr. Raman said treatment for at least 6 months may be necessary to achieve its benefits.
“We know that a sensitive measurement of heart function known as strain is abnormal well before complications like congestive heart failure and fatal arrhythmias occur in DMD. By impacting this earliest detectable change in heart function, we expect and hope to see even greater benefits with longer-term follow-up of these patients. Slowing the progression of heart disease should translate into improved quality of life for affected individuals and their families,” Dr. Raman said.
The study was funded by the Parent Project Muscular Dystrophy, the National Center for Advancing Translational Sciences and the National Institutes of Health. It was also inspired by Ryan Ballou, a 26-year-old DMD patient from Pittsburgh, who created fundraiser BallouSkies together with his father.
Dr. Raman is already preparing to launch a follow-up clinical trial sometime early 2015 in several sites across the country: Ohio State’s Wexner Medical Center/Nationwide Children’s Hospital, UCLA, the University of Colorado and the University of Utah. Subscribe to Muscular Dystrophy News, and follow us on Facebook and Twitter to stay updated.
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