Adolescent patients with obstructive hypertrophic cardiomyopathy (HCM) who received the drug mavacamten saw a significant improvement in left ventricular outflow tract (LVOT) gradient, a measure of blood flow obstruction in the heart, compared with those who received a placebo, according to a small study presented at the American College of Cardiology's Annual Scientific Session (ACC.26). The trial is the first to test mavacamten in patients younger than 18. This study was simultaneously published online in the New England Journal of Medicine at the time of presentation.
"These results are very encouraging," said Joseph William Rossano, MD, chief of cardiology at Children's Hospital of Philadelphia and the study's lead author. "Patients feel better, and their hearts look better."
HCM is a genetic disorder in which the heart muscle thickens, causing the heart chambers to become smaller and potentially reducing its ability to pump blood. In many cases, the thickened muscle blocks or reduces the flow of blood into the aorta from the left ventricle, known as obstructive HCM. Obstructive HCM can cause symptoms such as chest pain, dizziness, shortness of breath and swelling and lead to heart failure and death.
https://en.wikipedia.org/wiki/Mavacamten
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