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Efficacy of Mavacamten on functional capacity in hypertrophic cardiomyopathy: a meta-analysis, meta-regression, and
Ibrahim Khalil1, Anannya Sheikh2, Nabila Nur3
1Department of Medicine, Dhaka Medical College and Hospital, Dhaka, Bangladesh.
Insights
Mavacamten significantly reduces left ventricular outflow tract (LVOT) obstruction and NT-proBNP levels in patients with hypertrophic cardiomyopathy (HCM). However, its effect on exercise capacity requires further investigation, with no increased risk of serious adverse events observed.
Area of Science:
- Cardiology
- Pharmacology
- Clinical Trials
Background:
- Hypertrophic cardiomyopathy (HCM) is a genetic heart condition characterized by left ventricular hypertrophy.
- This hypertrophy can lead to left ventricular outflow tract (LVOT) obstruction, heart failure, and sudden cardiac death.
- Mavacamten is a novel cardiac myosin inhibitor designed to address the underlying mechanisms of HCM.
Purpose of the Study:
- To evaluate the efficacy and safety of Mavacamten in adults with HCM.
- To assess the impact of Mavacamten on left ventricular outflow tract (LVOT) gradients and NT-proBNP levels.
- To determine the effect of Mavacamten on exercise capacity and serious adverse events (SAEs).
Main Methods:
- A systematic literature search was conducted on PubMed, Cochrane Library, and ClinicalTrials.gov up to August 1, 2025.
- Six randomized controlled trials comparing Mavacamten to placebo in adult patients with HCM were included.
- Primary outcomes included changes in LVOT gradients; secondary outcomes included NT-proBNP levels, ejection fraction, pVO2, and SAEs. Data were analyzed using random-effects models with meta-regression and trial sequential analysis.
Main Results:
- Mavacamten significantly reduced post-exercise, post-Valsalva, and resting LVOT gradients (all P < 0.001).
- A significant decrease in NT-proBNP levels was observed (P < 0.00001).
- No significant changes in peak oxygen consumption (pVO2) were noted (P = 0.45), and the incidence of SAEs was comparable between groups (P = 0.66). Retrospective trial sequential analysis confirmed the robustness of the findings for LVOT gradient reduction.
Conclusions:
- Mavacamten demonstrates significant efficacy in reducing LVOT gradients and NT-proBNP levels in patients with HCM.
- The drug's impact on improving exercise capacity (pVO2) remains uncertain.
- Mavacamten appears safe, with no increased risk of serious adverse events compared to placebo.
Background:
Hypertrophic cardiomyopathy (HCM) is characterized by left ventricular hypertrophy, often leading to left ventricular outflow tract (LVOT) obstruction, heart failure, and sudden cardiac death. Mavacamten, a novel cardiac myosin inhibitor, targets the underlying mechanisms of HCM.
Methods:
We searched PubMed, Cochrane Library, and ClinicalTrials.gov up to 1 August 2025 and included six randomized controlled trials comparing Mavacamten to placebo in adults with HCM. The primary outcome was the change in LVOT gradients (post-exercise, post-Valsalva, and resting). Secondary outcomes included N-terminal pro-brain natriuretic peptide (NT-proBNP) levels, left ventricular ejection fraction, peak oxygen consumption (pVO2), and serious adverse events (SAEs). Data were analyzed using a random-effects model, with results reported as mean differences (MD) or risk ratios (RR) with 95% confidence intervals (CI). Meta-regression explored study-level moderators, and retrospective trial sequential analysis assessed the robustness of the findings.
Results:
Among 632 patients, Mavacamten significantly reduced LVOT gradients: post-exercise (MD: -38.28 mmHg, 95% CI: -45.92 to -30.63, P < 0.00001), post-Valsalva (MD: -59.02 mmHg, 95% CI: -82.75 to -35.29, P < 0.00001), and resting (MD: -44.50 mmHg, 95% CI: -66.49 to -22.51, P < 0.0001). NT-proBNP levels decreased significantly (MD: -617.26 ng/L, 95% CI: -772.43 to -462.09, P < 0.00001), but pVO2 showed no significant change (MD: 0.69 mL/kg/min, 95% CI: -1.12 to 2.50, P = 0.45). SAEs were comparable (RR: 1.17, 95% CI: 0.58-2.36, P = 0.66). Across primary outcomes, retrospective trial sequential analysis confirms that Mavacamten's reductions in LVOT gradients are conclusive, indicating robust evidence.
Conclusion:
Mavacamten effectively reduces LVOT gradients and NT-proBNP levels in HCM, but its impact on exercise capacity remains uncertain, with no increased risk of SAEs.
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