Clinical effectiveness of mavacamten on cardiac function and disease-related indicators in patients with obstructive hypertrophic cardiomyopathy

Objective This study aimed to compare the clinical effects of mavacamten and conventional pharmacotherapy on cardiac function and related disease indicators in patients with obstructive hypertrophic cardiomyopathy (oHCM). Methods A retrospective analysis was performed in patients with oHCM who were hospitalized between January 2024 and May 2025. The intervention group included 126 patients who received mavacamten for the first time, while the control group consisted of 134 patients treated with conventional medications. Baseline clinical characteristics, laboratory parameters, and imaging findings were collected. All patients were followed up for 30 weeks after discharge. Changes in relevant clinical indicators were recorded and compared at weeks 6 (W6), 18 (W18), and 30 (W30). Results A total of 222 patients (111 per group) completed the 30-week follow-up and were included in the final analysis. After 30 weeks of treatment, the resting left ventricular outflow tract (LVOT) peak gradient fell to 23.76 ± 4.09 mmHg in the intervention group but persisted at 78.49 ± 13.21 mmHg in the control group (t = 24.074, p < 0.001). Similarly, the Valsalva-provoked LVOT peak gradient decreased to 48.95 ± 11.10 mmHg versus 113.59 ± 14.92 mmHg in the control group (t = 21.147, p < 0.001). Left ventricular ejection fraction (LVEF) was 55.12% ± 4.85% in the intervention group and 56.78% ± 5.42% in the control group (p = 0.312), with 9.5% of mavacamten-treated patients experiencing LVEF decline to <50% (vs. 2.2% in controls). N-terminal pro-B-type natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin I (hs-cTnI) levels were significantly lower in the intervention group (p < 0.05). Conclusion Mavacamten indicates significant clinical association with improved outcomes in improving cardiac function and related disease indicators in patients with obstructive hypertrophic cardiomyopathy, with sustained reductions in both resting and provoked LVOT gradients.