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 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 cardiomyo-pathy 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.