Mavacamten treatment resulted in a 78% symptomatic response and 93% complete hemodynamic response, with significant reductions in LVOT gradient and NT-proBNP levels.
Does mavacamten improve symptomatic and hemodynamic responses in patients with hypertrophic obstructive cardiomyopathy?
In a real-world cohort of patients with hypertrophic obstructive cardiomyopathy, mavacamten therapy demonstrated high rates of symptomatic and hemodynamic improvement comparable to pivotal clinical trials.
Aims To provide real-world data on the symptomatic and hemodynamic response of the myosin inhibitor mavacamten in patients with hypertrophic obstructive cardiomyopathy (HOCM). Methods Patients with HOCM up-titrated to their final mavacamten dose were included. The final dose was defined as (i) 15 mg daily (or 5 mg for poor CYP2C19 metabolizers), (ii) a dose achieving a complete hemodynamic response (LVOT gradient 30 mmHg), or (iii) a lower dose limited by adverse effects. Final evaluation was performed 12 weeks after reaching the final dose. Symptomatic response was defined as ≥1 NYHA class improvement, and incomplete hemodynamic response as residual LVOT gradient ≥30 mmHg. Results 40 patients (56 ± 12 years, 78% male) were included. The LVOT gradient (rest: −25 ± 32 mmHg, p 0.001, Valsalva: −73 ± 56 mmHg, p 0.001) and NT-proBNP levels (−785 ± 1,122 ng/L, p 0.001) significantly decreased during a mean follow up of 184 ± 83 days. 78% had a symptomatic response and 93% were complete hemodynamic responders. Patients with no improvement in NYHA class had a lower e′ lat. (9 ± 3 cm/s vs. 6 ± 2 cm/s, p = 0.034) and less often baseline therapy with beta-blockers. Patients with incomplete hemodynamic response had a significantly higher baseline septum thickness (26.3 ± 4.9 mm vs. 19.1 ± 3.7 mm, p = 0.007) and higher LV-mass index (205 ± 63 mL/m 2 vs. 139 ± 31 mL/m 2 , p = 0.038). Absolute reduction of LVOT gradients was similar in patients with and without clinical or hemodynamic response. Conclusion Clinical and hemodynamic response to mavacamten was high in this real-world cohort and comparable to pivotal trial results. Incomplete response might be related to more severe baseline disease, which needs further study.
Seuthe et al. (2026) studied this question. Mavacamten treatment resulted in a 78% symptomatic response and 93% complete hemodynamic response, with significant reductions in LVOT gradient and NT-proBNP levels.