Evaluation of mavacamten in patients with hypertrophic cardiomyopathy

Aims We aimed to comprehensively assess the safety and efficacy of mavacamten in hypertrophic cardiomyopathy (HCM) patients. Methods A systematic review and meta-analysis was conducted, and efficacy [changes in postexercise left ventricular outflow tract (LVOT) gradient, left ventricular ejection fraction (LVEF), peak oxygen consumption (pVO2), Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS), and the proportion of patients exhibiting an improvement of at least one New York Heart Association (NYHA) functional class from baseline)], safety (total count of treatment-emergent adverse events and SAEs, as well as the proportion of patients experiencing at least one adverse event or SAE), and cardiac biomarkers (NT-proBNP and cTnI) outcomes were evaluated. Results We incorporated data from four randomized controlled trials, namely EXPLORER-HCM, VALOR-HCM, MAVERICK-HCM, and EXPLORER-CN. Mavacamten demonstrated significant efficacy in reducing the postexercise LVOT gradient by 49.44 mmHg (P = 0.0001) and LVEF by 3.84 (P < 0.0001) and improving pVO2 by 0.69 ml/kg/min (P = 0.4547), KCCQ CSS by 8.11 points (P < 0.0001), and patients with at least one NYHA functional class improvement from baseline by 2.20 times (P < 0.0001). Importantly, mavacamten increased 1.11-fold adverse events (P = 0.0184) 4.24-fold reduced LVEF to less than 50% (P = 0.0233) and 1.06-fold SAEs (P = 0.8631). Additionally, mavacamten decreased NT-proBNP by 528.62 ng/l (P < 0.0001) and cTnI by 8.28 ng/l (P < 0.0001). Conclusion Mavacamten demonstrates both safety and efficacy in patients with HCM, suggesting its potential as a promising therapeutic strategy for this condition. Further research is warranted to confirm these results and explore its long-term effects.