Abstract
Hypertrophic obstructive cardiomyopathy (HOCM) is a complex genetic cardiac disease that causes left ventricular hypertrophy and obstruction of the outflow tract. Mavacamten, a novel cardiac myosin inhibitor, has emerged as a potentially beneficial therapeutic option. This meta-analysis aimed to determine whether mavacamten is effective and safe for use in patients with HOCM. A systematic literature search was performed in PubMed and the Cochrane Central Register of Controlled Trials to identify randomized controlled trials (RCTs) that compared mavacamten to placebo in patients with HOCM. The primary objectives were changes in the gradients associated with the Valsalva maneuver and resting left ventricular outflow tract (LVOT). Alterations in the left atrial volume index (LAVI), left ventricular mass index (LVMI), and NT-proBNP level were secondary outcomes. Safety outcomes were also evaluated. Random effects models were used in the meta-analysis. Two RCTs comprising 332 patients were included. Mavacamten significantly reduced the Valsalva LVOT gradient (mean difference (MD) = -54.94 mmHg; 95% CI: -70.32, -39.56; P = 0.13) and resting LVOT gradient (MD = -42.44 mmHg; 95% CI: -67.52, -17.36; P<0.001) compared to placebo. Significant improvements were also observed in LAVI (MD = -7.18 mL/m²; 95% CI: -11.00, -3.37; P = 0.24) and NT-proBNP levels (RR = 0.58; 95% CI: 0.39, 0.84; P<0.001). LVMI showed a trend toward reduction (MD = -19.15 g/m²; 95% CI: -41.98, 3.69; P<0.001). Mavacamten demonstrated a favorable safety profile with few reported adverse events. This meta-analysis aimed to demonstrate the efficacy and short-term safety of mavacamten in patients with HOCM. Considerable improvement was observed in the LVOT gradients, cardiac remodeling measures, and indicators of cardiac stress when mavacamten was administered. Based on this data, mavacamten appears to offer the potential for a paradigm shift in the management of HOCM. However, studies conducted over an extended period are required to validate its long-term effectiveness and safety profile.