A recent long-term study published in the European Journal of Neurology found that starting heart medications (perindopril and bisoprolol) early in boys with Duchenne muscular dystrophy (DMD) did not prevent the heart’s decline over five years when compared to starting the medications a few years later. However, there were signs that starting the medications earlier might still be helpful.
After five years, more of the boys who had started taking the medications later had passed away, had reduced heart function or were taking additional heart medications compared to the group who began medications earlier. This supports the current recommendation to start heart medications early as a preventative measure in DMD.
Perindopril and bisoprolol were well tolerated, with no major side effects or issues that led to participants stopping treatment.
Unfortunately, the findings of this study are not clear-cut because the study’s results were influenced by the small number of participants, the lack of a placebo group and the use of other heart medications. For example, most patients were receiving steroids for muscle strengthening, and steroids are known to delay the onset of cardiomyopathy.
Read more about DMD treatment and care
The goal of this study was to determine if starting heart medications earlier would be more effective than waiting a few years. In a previous study, no differences were found between boys taking the medications and those on a placebo after three years.
Out of the original 75 boys in the first study, 65 boys (average age 16) returned for the follow-up study. Researchers mainly measured how well their hearts were pumping blood, using a test called left ventricular ejection fraction (LVEF%).
Results showed that heart function declined in both groups, whether they started the medications early or later. But a closer look at the results suggested that preventative (or prophylactic) treatment might still be beneficial.
“Various trends favoring the introduction of prophylactic therapy were evident in sub-group analyses, supporting current DMD standards of care recommendations to introduce heart medications prophylactically,” the study’s authors said.
The study also highlighted the challenges of studying heart health in DMD and shows that future research needs to consider many factors when examining treatment options.
Sign up here to get the latest news, perspectives, and information about DMD sent directly to your inbox. Registration is free and only takes a minute.
