{Reference Type}: Journal Article
{Title}: Evaluation of mavacamten in patients with hypertrophic cardiomyopathy.
{Author}: Liao HL;Liang Y;Liang B;
{Journal}: J Cardiovasc Med (Hagerstown)
{Volume}: 25
{Issue}: 7
{Year}: 2024 Jul 1
{Factor}: 2.43
{DOI}: 10.2459/JCM.0000000000001638
{Abstract}: <B>OBJECTIVE: </B>We aimed to comprehensively assess the safety and efficacy of mavacamten in hypertrophic cardiomyopathy (HCM) patients.<BR><B>METHODS: </B>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 (pVO 2 ), 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.<BR><B>RESULTS: </B>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 pVO 2 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).<BR><B>CONCLUSIONS: </B>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.