Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials

Abstract Background Mavacamten, an allosteric myosin inhibitor, is considered to be a promising drug for the treatment of hypertrophic cardiomyopathy (HCM). This meta-analysis aimed to explore the safety and efficacy of mavacamten in HCM patients. Main body A total number of 539 patients were enroll...

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Main Authors: Mehrdad Rabiee Rad, Ghazal Ghasempour Dabaghi, Danial Habibi
Format: Article
Language:English
Published: SpringerOpen 2023-01-01
Series:The Egyptian Heart Journal
Subjects:
Online Access:https://doi.org/10.1186/s43044-023-00328-7
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author Mehrdad Rabiee Rad
Ghazal Ghasempour Dabaghi
Danial Habibi
author_facet Mehrdad Rabiee Rad
Ghazal Ghasempour Dabaghi
Danial Habibi
author_sort Mehrdad Rabiee Rad
collection DOAJ
description Abstract Background Mavacamten, an allosteric myosin inhibitor, is considered to be a promising drug for the treatment of hypertrophic cardiomyopathy (HCM). This meta-analysis aimed to explore the safety and efficacy of mavacamten in HCM patients. Main body A total number of 539 patients were enrolled in four randomized clinical trials. The mean age of patients was 57.9 years and was followed for 29.3 weeks. Pooled analysis showed a significant improvement in clinical response (Log OR = 0.65; p = 0.01) and the number of patients with a reduction of ≥ 1 NYHA function class (Log OR = 0.64, p = 0.00). It was found that mavacamten did not significantly affect the Kansas City Cardiomyopathy Questionnaire (KCCQ) (SMD = 0.43, p = 0.08), peak oxygen uptake (PVO2) (SMD = 0.24, p = 0.42), and ejection fraction (EF) (SMD = − 0.65, p = 0.13) as compared with placebo. However, KCCQ (SMD = 0.65, 95% CI 0.44–0.87) and PVO2 (SMD = 0.49, 95% CI 0.24–0.74) improvements were statically significant in the hypertrophic obstructive cardiomyopathy subgroup (HOCM), and a significant decrease in EF (SMD = -− 1.14, 95% CI − 1.86 to − 0.42) was found in the HOCM subgroup. No significant difference was observed in the incidence rate of serious adverse events between mavacamten and placebo group (Log OR = − 0.23, p = 0.56). Conclusions Mavacamten proved to be effective and well-tolerated for the treatment of HCM. Mavacamten improved the signs and symptoms of HOCM and decreased EF in these patients without serious adverse events in the clinical trials.
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spelling doaj.art-809ca1fd34a8481ca09c384b5b7e98232023-01-15T12:15:10ZengSpringerOpenThe Egyptian Heart Journal2090-911X2023-01-017511710.1186/s43044-023-00328-7Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trialsMehrdad Rabiee Rad0Ghazal Ghasempour Dabaghi1Danial Habibi2School of Medicine, Isfahan University of Medical ScienceSchool of Medicine, Isfahan University of Medical ScienceDepartment of Biostatistics and Epidemiology, Faculty of Health, Isfahan University of Medical ScienceAbstract Background Mavacamten, an allosteric myosin inhibitor, is considered to be a promising drug for the treatment of hypertrophic cardiomyopathy (HCM). This meta-analysis aimed to explore the safety and efficacy of mavacamten in HCM patients. Main body A total number of 539 patients were enrolled in four randomized clinical trials. The mean age of patients was 57.9 years and was followed for 29.3 weeks. Pooled analysis showed a significant improvement in clinical response (Log OR = 0.65; p = 0.01) and the number of patients with a reduction of ≥ 1 NYHA function class (Log OR = 0.64, p = 0.00). It was found that mavacamten did not significantly affect the Kansas City Cardiomyopathy Questionnaire (KCCQ) (SMD = 0.43, p = 0.08), peak oxygen uptake (PVO2) (SMD = 0.24, p = 0.42), and ejection fraction (EF) (SMD = − 0.65, p = 0.13) as compared with placebo. However, KCCQ (SMD = 0.65, 95% CI 0.44–0.87) and PVO2 (SMD = 0.49, 95% CI 0.24–0.74) improvements were statically significant in the hypertrophic obstructive cardiomyopathy subgroup (HOCM), and a significant decrease in EF (SMD = -− 1.14, 95% CI − 1.86 to − 0.42) was found in the HOCM subgroup. No significant difference was observed in the incidence rate of serious adverse events between mavacamten and placebo group (Log OR = − 0.23, p = 0.56). Conclusions Mavacamten proved to be effective and well-tolerated for the treatment of HCM. Mavacamten improved the signs and symptoms of HOCM and decreased EF in these patients without serious adverse events in the clinical trials.https://doi.org/10.1186/s43044-023-00328-7Hypertrophic cardiomyopathyMavacamtenHCMHOCM
spellingShingle Mehrdad Rabiee Rad
Ghazal Ghasempour Dabaghi
Danial Habibi
Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
The Egyptian Heart Journal
Hypertrophic cardiomyopathy
Mavacamten
HCM
HOCM
title Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
title_full Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
title_fullStr Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
title_full_unstemmed Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
title_short Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials
title_sort safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy a systematic review and meta analysis of randomized clinical trials
topic Hypertrophic cardiomyopathy
Mavacamten
HCM
HOCM
url https://doi.org/10.1186/s43044-023-00328-7
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