Mavacamten did not significantly improve KCCQ score (MD 2.12; 95% CI -0.98 to 5.22) or peak oxygen consumption compared to placebo in patients with non-obstructive hypertrophic cardiomyopathy.
Meta-Analysis (n=639)
Does mavacamten improve symptoms, functional capacity, or health status in patients with non-obstructive hypertrophic cardiomyopathy compared to placebo?
In patients with non-obstructive hypertrophic cardiomyopathy, mavacamten does not significantly improve symptoms or functional capacity compared to placebo, and is associated with an increased risk of atrial fibrillation.
Estimación del efecto: MD 2.12 (95% CI -0.98 to 5.22)
Non-obstructive hypertrophic cardiomyopathy (nHCM) remains a therapeutic challenge, with limited pharmacological options beyond symptomatic management. Mavacamten, a selective allosteric cardiac myosin inhibitor, has shown benefit in obstructive HCM, but its role in nHCM is uncertain. A systematic search of PubMed, Embase, CENTRAL, and ClinicalTrials.gov was conducted up to November 2025 for placebo-controlled randomized controlled trials (RCTs) enrolling patients with nHCM. Outcomes included Kansas City Cardiomyopathy Questionnaire (KCCQ) score, peak oxygen consumption (pVO 2 ), New York Heart Association (NYHA) functional class, and treatment-emergent adverse events (TEAEs). Pooled mean differences (MDs) and risk ratios (RRs) were estimated using random- or fixed-effects models based on heterogeneity. Two RCTs (n = 639) met inclusion criteria. Mavacamten showed a nonsignificant improvement in KCCQ (MD: 2.12; 95% CI: –0.98 to 5.22; I 2 = 0%) and peak oxygen consumption (MD: 0.36 mL/kg/min; 95% CI: –0.20 to 0.92; I 2 = 0%). No significant difference was observed in NYHA class improvement (RR: 1.15; 95% CI: 0.93 to 1.43; I 2 = 0%). TEAE incidence was similar between groups (RR: 1.11; 95% CI: 0.34 to 3.58; I 2 = 40.8%). Atrial fibrillation occurred more frequently with mavacamten (RR: 1.41; 95% CI: 1.28 to 1.56), whereas palpitations and dizziness did not differ significantly. Mavacamten does not significantly enhance symptoms, functional capacity, or health status in nHCM, though safety is comparable to placebo. Ongoing monitoring for arrhythmias and reduced ejection fraction is essential. Larger, longer-term studies are needed to clarify its role in this population.
Abdullah et al. (Thu,) conducted a meta-analysis in Non-obstructive hypertrophic cardiomyopathy (nHCM) (n=639). Mavacamten vs. Placebo was evaluated on Kansas City Cardiomyopathy Questionnaire (KCCQ) score (MD 2.12, 95% CI -0.98 to 5.22). Mavacamten did not significantly improve KCCQ score (MD 2.12; 95% CI -0.98 to 5.22) or peak oxygen consumption compared to placebo in patients with non-obstructive hypertrophic cardiomyopathy.