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

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 (PVO(2)) (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 PVO(2) (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. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s43044-023-00328-7.