Efficacy and safety of mavacamten for the treatment of hypertrophic cardiomyopathy: an updated systematic review and meta-analysis of randomized controlled trials

Abstract

The efficacy and safety profile of mavacamten, a cardiac myosin inhibitor for the treatment of hypertrophic cardiomyopathy (HCM) is not well-established, prompting the need for an updated meta-analysis. The authors conducted an extensive search across multiple electronic databases, including Embase, MEDLINE (via Pubmed), and CENTRAL, to identify randomized controlled trials (RCTs) assessing the efficacy and safety of mavacamten in HCM. Review Manager 5.4 (Revman) was employed to pool risk ratios (RR) and mean differences (MD). Our literature search yielded 4 RCTs with a total of 503 patients. Mavacamten was found to be associated with higher rates of greater than or equal to 1 New York Heart Association (NYHA) class improvement (RR 2.20, 95% CI: 1.48-3.28; I²=51%) and change from baseline in the Kansas City Cardiomyopathy Questionnaire- Clinical Summary Score (KCCQ-CSS) (MD 7.50, 95% CI: 3.44-11.55; I² =50%). Mavacamten was also associated with improved resting left ventricular outflow tract (LVOT) gradient (MD -38.33, 95% CI: -49.38 to -27.28; I² =75%), Valsalva LVOT gradient (MD -48.08, 95% CI: -62.21 to -33.96; I² =78%), post-exercise LVOT gradient (MD -37.1, 95% CI: -44.37 to -29.84; I² =0%), LVMI (MD -16.91, 95% CI: -28.29 to -5.54; I² =88%), and lower rates of septal reduction therapy (SRT) (RR 0.30, 95% CI: 0.22-0.40; I² =0%). There were no significant differences between mavacamten and placebo regarding the composite functional outcome, greater than or equal to 1 treatment-emergent adverse event, greater than or equal to 1 serious adverse event, and atrial fibrillation. The authors; findings suggest that mavacamten contributes to improvements in NYHA class, KCCQ-CSS scores, and LVOT gradients while reducing the incidence of SRT in patients with HCM.

Keywords: HOCM; hypertrophic cardiomyopathy; hypertrophic obstructive cardiomyopathy; mavacamten.

Figure 1

Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) flowchart. RCT, randomized controlled trial.

Figure 2

Forest plot of the effect of mavacamten vs placebo on ≥1 New York Heart Association (NYHA) class improvement. HCM, hypertrophic cardiomyopathy.

Figure 3

Forest plot of the effect of mavacamten vs placebo on change from baseline in Kansa City Cardiomyopathy Questionnaire Score (KCCQ-CSS). HCM, hypertrophic cardiomyopathy.