A Systematic Review and Meta-analysis of Efficacy and Safety of Mavacamten for the Treatment of Hypertrophic Cardiomyopathy.

Background: Hypertrophic cardiomyopathy (HCM) is a common hereditary cardiomyopathy. Mavacamten, a first-in-class cardiac myosin inhibitor, is considered to be a specific drug for the treatment of HCM. This meta-analysis aimed to assess the efficacy and safety of mavacamten in patients with HCM.

Methods: PubMed, Cochrane Library, Embase and Clinical Trials.gov databases were searched from inception to February 6, 2024 for randomized controlled trials (RCTs) which compared the efficacy and safety between mavacamten and placebo in treating HCM.

Results: Six RCTs involving 732 patients were included in this meta-analysis. This meta-analysis showed that mavacamten improved the New York Heart Association (NYHA) function class [risk ratios (RR): 2.21, 95% confidence interval (CI): 1.48 to 3.30, = 0.00001], Clinical Summary Score of the Kansas City Cardiomyopathy Questionnaire (KCCQ-CSS) scores [mean difference (MD): 9.33, 95% CI: 7.09 to 11.57, < 0.00001] and composite functional end point (RR: 1.86, 95% CI: 1.25 to 2.78, = 0.002). Meanwhile, mavacamten decreased N-terminal pro-B-type natriuretic peptide (NT-proBNP) (MD: -492.28, 95% CI: -611.55 to -373.02, < 0.00001), cardiac troponin I (cTnI) (MD: -14.58, 95% CI: -26.98 to -2.17, = 0.02) and Valsalva left ventricular outflow tract (LVOT) gradient (MD: -57.96, 95% CI: -82.15 to -33.78, < 0.00001). The results for the incidence of ≥1 total emergent adverse event (TEAE) and ≥1 serious adverse event (SAE) showed that there was no significant difference between both groups (RR: 1.9, 95% CI: 0.97 to 1.24, = 0.16) (RR: 1.06, 95% CI: 0.46 to 2.44, = 0.90).

Conclusions: Mavacamten has great efficacy for the treatment of HCM. Meanwhile, mavacamten did not increase the incidence of adverse events or serious adverse events.