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Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): A randomised, double-blind, placebo-controlled, phase 3 trial

The Lancet Sep 04, 2020

Olivotto I, Oreziak A, Barriales-Villa R, et al. - This phase 3, randomised, double-blind, placebo-controlled trial (EXPLORER-HCM) was undertaken to determine whether mavacamten, a first-in-class cardiac myosin inhibitor, affords a safe and efficacious treatment option for patients with symptomatic obstructive hypertrophic cardiomyopathy. This inquiry was performed in 68 clinical cardiovascular centres in 13 countries. Participants were patients suffering from hypertrophic cardiomyopathy with an left ventricular outflow tract (LVOT) gradient of 50 mm Hg or greater and NYHA class II–III symptoms. These patients were assigned (1:1) to receive mavacamten (starting at 5 mg) or placebo for 30 weeks. Findings showed that improvements in exercise capacity, LVOT obstruction, NYHA functional class, and health status were conferred by treatment with mavacamten in patients with obstructive hypertrophic cardiomyopathy. As per results, disease-specific treatment is beneficial for this condition.

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