Mavacamten treatment in a real-world cohort achieved the primary effectiveness endpoint of reduced LVOTG or improved NYHA class in 90% of patients with obstructive hypertrophic cardiomyopathy.
Cohort (n=82)
Sí
Does mavacamten improve hemodynamics and symptoms in real-world adult patients with symptomatic obstructive hypertrophic cardiomyopathy?
Real-world data confirms the effectiveness of mavacamten in reducing LVOTG and improving symptoms in oHCM, while highlighting the need for careful monitoring due to transient LVEF reductions and variable response patterns.
INTRODUCTION Mavacamten has demonstrated efficacy and safety in randomized trials for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM), but detailed real-world data in clinically complex populations remain limited. METHODS We evaluated the effectiveness, response patterns, safety and implementation challenges of mavacamten in a multicenter cohort (SWISS-MAVA) of adult patients with oHCM treated without CYP2C19 genotyping. The primary outcome was reduction of provocable (Valsalva) left ventricular outflow tract gradient (LVOTGval) ≤ 30 mmHg, or LVOTGval≤50 mmHg with ≥1 NYHA class improvement at last follow-up. RESULTS Eighty-two patients were included (age 59.5 ± 13.4 years, 63% male) and followed for 52.4 ± 21.4 weeks. Mavacamten reduced LVOTGval from 8262 to 112 to 206 to 25 mmHg, NT-proBNP from 537273-1279 to 14277-303 ng/L, E/e' from 14.5 ± 6.6 to 10.8 ± 4.5 and left atrial volume indexed from 44.7 ± 12.5 to 37.2 ± 11.2 mL/m2 (p < 0.001). Mean left ventricular ejection fraction (LVEF) decreased (66.7 ± 5.4% to 62.6 ± 5.8%, (p < 0.001)) but remained preserved overall. The primary endpoint was achieved in 74/82 patients (90%), with 62/82 (76%) responding within 6 months. Twelve patients (15%) showed a super-hemodynamic response requiring protocol-mandated dose reduction, prolonging titration by 3.5 ± 2 months. Transient LVEF<50% occurred in 5 patients (6%; 5.9/100 patient-years), all reversible after treatment interruption; in 4/5 cases, a potential cofactor was identified. New-onset atrial fibrillation occurred in 6 patients (7.3%; 7.1/100 patient-years). Weight loss was observed in some obese/overweight responders, with transitions to lower BMI categories. CONCLUSIONS These data support the effectiveness and safety of mavacamten in real-world practice while highlighting clinically relevant heterogeneity in response patterns, titration burden, and adverse-event susceptibility.
Maurizi et al. (Wed,) conducted a cohort in obstructive hypertrophic cardiomyopathy (oHCM) (n=82). Mavacamten was evaluated on Reduction of provocable (Valsalva) left ventricular outflow tract gradient (LVOTGval) ≤ 30 mmHg, or LVOTGval ≤ 50 mmHg with ≥ 1 NYHA class improvement at last follow-up. Mavacamten treatment in a real-world cohort achieved the primary effectiveness endpoint of reduced LVOTG or improved NYHA class in 90% of patients with obstructive hypertrophic cardiomyopathy.