Key result
Cardiac myosin inhibitors increase odds of functional class improvement ~310% vs placebo in symptomatic HCM.
Why the study?
Hypertrophic cardiomyopathy lacks highly effective pharmacologic treatments, and updated efficacy data on cardiac myosin inhibitors were needed.
Do cardiac myosin inhibitors improve functional class and symptoms in patients with symptomatic hypertrophic cardiomyopathy?
Meta-Analysis (n=826)
Do cardiac myosin inhibitors improve functional class and symptoms in patients with symptomatic hypertrophic cardiomyopathy?
Cardiac myosin inhibitors significantly improve functional class, symptoms, and LVOT gradients in patients with symptomatic hypertrophic cardiomyopathy without increasing serious adverse events.
Answers are grounded in this paper and cite their sources.
Supports CMI for NYHA improvement in symptomatic HCM; confirms efficacy on symptoms and gradients across six RCTs.
Pedro Henrique Tavares Fogaça , Gabriel Grando Alves , Camila Sales Sales Fagundes , Fernanda Lavarda Scheinpflug , João Candido Tavares Fogaça , Mateus Diniz Marques DOI: 10.36660/ijcs.20250092 This study evaluates the efficacy of cardiac myosin inhibitors (CMI), which appear to be an innovative alternative for hypertrophic cardiomyopathy (HCM), a disease lacking highly effective pharmacologic treatments. To provide updated efficacy data on CMI (aficamten or mavacamten) in symptomatic HCM. We searched PubMed, Embase, and Cochrane databases for randomized controlled trials comparing CMI to placebo in symptomatic HCM, reporting: (1) New York Heart Association (NYHA) functional class improvement; (2) change from baseline in Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS); (3) percent change from baseline in left ventricular outflow tract (LVOT) gradient at rest and after Valsalva maneuver; and (4) serious adverse events. Heterogeneity was examined with I 2 statistics, and p values < 0.05 indicated sta tistical significance. Continuous endpoints were analyzed using pooled mean difference (MD), and binary endpoints were analyzed using odds ratios (OR), both with 95% confidence intervals (CI). We included six RCTs with 826 patients, 443 of whom received CMI. Compared to placebo, NYHA class improvement was more frequent in patients receiving CMI (OR: 4.10; 95% CI: 2.79 to 6.02; p < 0.00001), with significant differences in KCCQ-CSS change (MD: 7.15; 95% CI: 4.21 to 10.10; p < 0.00001), as well as in LVOT gradient change at rest (MD: −38.25; 95% CI: −46.76 to −29.74; p < 0.00001) and after Valsalva maneuver (MD: −46.49; 95% CI: −54.70 to −38.27; p < 0.00001). There were no significant differences in serious adverse events (OR: 0.64; 95% CI: 0.31 to 1.31; p = 0.22). CMI shows greater efficacy than placebo in treating symptomatic HCM.
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Fogaça et al. (2026) conducted a meta-analysis in Symptomatic Hypertrophic Cardiomyopathy (n=826). Cardiac myosin inhibitors (aficamten or mavacamten) vs. Placebo was evaluated on New York Heart Association (NYHA) functional class improvement (OR 4.10, 95% CI 2.79 to 6.02, p=< 0.00001). Cardiac myosin inhibitors increased the likelihood of functional class improvement compared to placebo in symptomatic hypertrophic cardiomyopathy.
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