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

The Lancet May 22, 2021

Spertus JA, Fine JT, Elliott P, et al. - Researchers investigated how mavacamten, a first-in-class cardiac myosin inhibitor, could impact health status—ie, symptoms, physical and social function, and quality of life in patients suffering from obstructive hypertrophic cardiomyopathy. This work represents a health status analysis of EXPLORER-HCM, a phase 3, double-blind, randomised, placebo-controlled trial. Randomization was used to assign (1:1) adult patients (≥18 years) with symptomatic obstructive hypertrophic cardiomyopathy (gradient ≥50 mm Hg and New York Heart Association class II–III) to receive mavacamten or placebo for 30 weeks, followed by an 8-week washout period. A markedly improved health status of participants was shown to be conferred by mavacamten vs placebo, with a low number required to treat for marked improvement. Since the main objectives of treatment are to improve symptoms, physical and social function, and quality of life, thus, mavacamten affords a new potential option for achieving these goals.

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