open access

Online first
Review paper
Published online: 2021-07-09
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Mavacamten — a new disease-specific option for pharmacological treatment of symptomatic patients with hypertrophic cardiomyopathy

Piotr Pysz, Renata Rajtar-Salwa, Grzegorz Smolka, Iacopo Olivotto, Wojciech Wojakowski, Paweł Petkow-Dimitrow
DOI: 10.33963/KP.a2021.0064
·
Pubmed: 34268723

open access

Online first
Review article
Published online: 2021-07-09

Abstract

Current pharmacotherapy for hypertrophic cardiomyopathy (HCM) is not disease-specific and has suboptimal efficacy, often necessitating interventional treatment. EXPLORER-HCM was a phase 3, randomized, double-blind, placebo-controlled multicenter clinical trial investigating effects of mavacamten, a first-in-class selective cardiac myosin inhibitor, in patients with HCM, left ventricular outflow obstruction (LVOTO) and New York Heart Association (NYHA) class II or III symptoms. The primary end-point was defined as either a ≥1.5 ml/kg/min increase in peak oxygen consumption (pVO2) and ≥1 NYHA class reduction or a ≥3.0 ml/kg/min pVO2 increase without NYHA class worsening. Secondary end-points evaluated changes in post-exercise LVOT gradient, pVO2, NYHA class, Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS), and Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness-of-Breath subscore (HCMSQ-SoB). A total of 251 patients were randomized to receiving mavacamten or placebo. The primary and all secondary end-points were met significantly more frequently in the mavacamten arm versus placebo. The safety profile of mavacamten was similar to that of placebo. In conclusion, disease-specific treatment with mavacamten in patients with obstructive HCM led to reduced LVOTO and improvement in both objective functional parameters and patient-related health status.

Abstract

Current pharmacotherapy for hypertrophic cardiomyopathy (HCM) is not disease-specific and has suboptimal efficacy, often necessitating interventional treatment. EXPLORER-HCM was a phase 3, randomized, double-blind, placebo-controlled multicenter clinical trial investigating effects of mavacamten, a first-in-class selective cardiac myosin inhibitor, in patients with HCM, left ventricular outflow obstruction (LVOTO) and New York Heart Association (NYHA) class II or III symptoms. The primary end-point was defined as either a ≥1.5 ml/kg/min increase in peak oxygen consumption (pVO2) and ≥1 NYHA class reduction or a ≥3.0 ml/kg/min pVO2 increase without NYHA class worsening. Secondary end-points evaluated changes in post-exercise LVOT gradient, pVO2, NYHA class, Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS), and Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness-of-Breath subscore (HCMSQ-SoB). A total of 251 patients were randomized to receiving mavacamten or placebo. The primary and all secondary end-points were met significantly more frequently in the mavacamten arm versus placebo. The safety profile of mavacamten was similar to that of placebo. In conclusion, disease-specific treatment with mavacamten in patients with obstructive HCM led to reduced LVOTO and improvement in both objective functional parameters and patient-related health status.

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Keywords

hypertrophic cardiomyopathy, left ventricular outflow obstruction, mavacamten

About this article
Title

Mavacamten — a new disease-specific option for pharmacological treatment of symptomatic patients with hypertrophic cardiomyopathy

Journal

Kardiologia Polska (Polish Heart Journal)

Issue

Online first

Article type

Review paper

Published online

2021-07-09

DOI

10.33963/KP.a2021.0064

Pubmed

34268723

Keywords

hypertrophic cardiomyopathy
left ventricular outflow obstruction
mavacamten

Authors

Piotr Pysz
Renata Rajtar-Salwa
Grzegorz Smolka
Iacopo Olivotto
Wojciech Wojakowski
Paweł Petkow-Dimitrow

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