Early treatment with ACE inhibitors improves cardiac outcomes in patients with Becker’s muscular dystrophy

There is no consensus on when to introduce cardioprotective therapy in patients with Becker’s muscular dystrophy (BMD), and practice varies widely. A new study published in the Journal of Neuromuscular Diseases concludes that starting treatment with an ACE inhibitor as soon as heart function falls below the lower limit of the normal range reduces the risk of developing severe cardiomyopathy and heart failure, which are common findings in BMD.

The prevention of end-stage heart failure, one of the most common causes of death in patients with BMD, is at the heart of their management. BMD is a form of muscular dystrophy related to Duchenne muscular dystrophy (DMD) in that both result from a mutation in the dystrophin gene, but with a milder course in the case of BMD. The latest practice guidelines from the American College of Cardiology / American Heart Association recommend prescribing an angiotensin converting enzyme (ACE-i) inhibitor to patients with non-ischemic cardiomyopathy when the ventricular ejection fraction left (LVEF) falls below 40%. However, there is no consensus on when to start treatment in patients with BMD.

“Although there is an abundant literature showing a beneficial effect of cardioprotective treatments such as ACE inhibitors in DMD, until now there was only limited evidence of a similar effect in BMD. “, explained principal investigator Karim Wahbi, MD, PhD, AP-HP, Myologie Institut; Cochin Hospital; University of Paris; and Inserm, UMRS, Paris, France. “As a result, there is currently no consensus among experts in the field of neuromuscular cardiomyopathies. And there remains a great variability regarding the use of ACE inhibitors in BMD from one country to another and center to center Should this treatment be initiated when cardiac function is normal as in DMD? Or when overt heart failure is present (left ventricular ejection less than 40%) as recommended in the general population? Or somewhere in between? We wanted to fill that gap. “

The researchers set out to determine whether starting treatment with an ACE inhibitor earlier than recommended by practice guidelines published by professional societies improves long-term cardiac outcomes in patients with BMD. They retrospectively analyzed data from 183 patients enrolled in a French multicenter BMD registry between January 1990 and April 2019 using a complex statistical analysis called propensity score analysis. They found that long-term cardiac outcomes in patients with BMD were significantly better when treatment with an ACE inhibitor was introduced earlier than recommended in current practice guidelines.

Among the patients having early treatment (when LVEF was less than 50%) versus conventional treatment (when LVEF was less than 40%), less than 4% were hospitalized for the management of heart failure compared to 11 , 8% who were treated conventionally and 17.6% versus 29.4% had a decrease in LVEF of less than 35%.

The magnitude of benefit conferred by ACE inhibitors in this study of patients with BMD is similar to that seen in a previous randomized study of patients with DMD, which reinforces their high efficacy in preventing myocardial dysfunction. , especially when introduced early in the course. of disease.

“This is the first study to show that treatment with an ACE inhibitor can benefit long-term heart health in patients with BMD, by reducing the risk of developing severe cardiomyopathy and heart failure. cardiac. In addition, it provides important information on the best time to start ACE inhibitors, as soon as heart function drops below the lower limit of the normal range, ”noted Dr Wahbi. We hope that these results will translate into greater and earlier use of ACE inhibitors in patients with BMD worldwide. “