Key result
Real-world use of mavacamten in 50 patients with obstructive hypertrophic cardiomyopathy over one year improved clinical parameters, with only 2 patients requiring temporary discontinuance for LVEF <50%.
Why the study?
Does mavacamten improve echocardiographic parameters and symptoms in real-world patients with obstructive hypertrophic cardiomyopathy?
Observational (n=50)
No
Does mavacamten improve echocardiographic parameters and symptoms in real-world patients with obstructive hypertrophic cardiomyopathy?
Real-world experience confirms that mavacamten is safe and effective for obstructive hypertrophic cardiomyopathy, with significant improvements in LVOT obstruction and symptoms over one year.
May support mavacamten in real-world oHCM; hypothesis-generating and requires larger randomized confirmation.
We describe real-world use of mavacamten in 50 patients with oHCM. Consistent with EXPLORER-HCM and VALOR-HCM, we report significant improvement in wall thickness, mitral regurgitation, left ventricular outflow tract obstruction and NYHA class. Moreover, in our center’s experience, neither arrhythmia burden, nor contractility have worsened in the vast majority of patients: We note a clinically insignificant mean decrease in left ventricular ejection fraction (LVEF), with only two patients requiring temporary mavacamten discontinuance for LVEF < 50%. Adverse events were rare, unrelated to mavacamten itself, and seen solely in patients with disease too advanced to have been represented in clinical trials. Our multidisciplinary pathway enabled us to provide a large number of patients with a novel closely-monitored therapeutic within just a few months of commercial availability. These data lead us to conclude that mavacamten, as a first-in-class cardiac myosin inhibitor, is safe and efficacious in real-world settings.
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Kim et al. (2023) conducted an observational in Obstructive Hypertrophic Cardiomyopathy (oHCM) (n=50). Mavacamten was evaluated. Real-world use of mavacamten in 50 patients with obstructive hypertrophic cardiomyopathy over one year improved clinical parameters, with only 2 patients requiring temporary discontinuance for LVEF <50%.
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