Researchers study benefits of treating young adults with hypertrophic cardiomyopathy before physical symptoms develop

A 3D image of a heart is shown against a blue medical backdrop

As researchers learn more about genetic associations with hypertrophic cardiomyopathy, a condition that affects about one in 500 adults, they are testing personalized approaches to support patients at risk for future cardiovascular events. While relatively uncommon, adverse events may include an irregular heart rhythmheart failure, or sudden cardiac death as a result of the heart thickening or becoming enlarged over time. An NHLBI-supported study, which published in Nature Medicine, found that young adults with heritable forms of the heart condition responded well to treatment before physical symptoms, like increased muscle thickness or inflammation, developed.  

Out of 178 adults who participated in the clinical research trial, 88 were assigned to the treatment group and received valsartan, a drug used to treat high blood pressure and heart failure. Ninety were in the control group and received a placebo. Over a two-year period, adults in the treatment group tolerated the drug and were less likely to experience physical changes associated with hypertrophic cardiomyopathy. The authors recommend future studies to help researchers assess the effectiveness and timing of this type of early-stage treatment approach.  

“We look forward to following this research in larger trials that study the immediate and long-term impacts of this type of targeted treatment,” said Kristin Burns, M.D., a study coauthor and chief of the Heart Development and Structural Diseases branch within the Division of Cardiovascular Sciences at the NHLBI.