Mavacamten initiated in 77.7% HOCM patients reduced LVOT gradient and NT-pro-BNP significantly by 4 weeks, improving NYHA III/IV from 50% to 11% at 20 weeks.
Does mavacamten improve LVOT gradients, biomarkers, and symptoms in patients with hypertrophic obstructive cardiomyopathy in a real-world setting?
Early real-world registry data confirms that mavacamten significantly reduces LVOT gradients and improves NYHA functional class in patients with hypertrophic obstructive cardiomyopathy, with an acceptable safety profile.
Absolute Event Rate: 0% vs 0%
Abstract Background Hypertrophic obstructive cardiomyopathy (HOCM) is associated with a high burden of heart failure. Myosin-inhibitor therapy targets the underlying pathophysiology of hypercontractility in HOCM, offering the potential for causal treatment and enhanced clinical outcomes. However, comprehensive large real-world data on HOCM management remain limited. Methods The ongoing multicenter German REDUCE-Registry (Registry for Patients with Diagnosed Hypertrophic Obstructive Cardiomyopathy Evaluated for Myosin-Inhibitor Therapy) involves seven cardiomyopathy referral centers across Germany. Patients presenting with HOCM since May 2023 are consecutively included. After informed consent, data from baseline and six-monthly follow-ups were collected. For patients receiving myosin-inhibitor therapy, additional data on efficacy (NYHA class, LVOT gradient, biomarkers) and adverse events were gathered during four-weekly follow-ups. Results A total of 200 HOCM patients (mean age 60.0 ± 12.0 years; 58% male) were enrolled thus far. Arterial hypertension was the most common comorbidity. Sarcomeric gene variants were found in 13.6% of patients, and 12.2% had a cardiac implantable electronic device (CIED). Baseline echocardiography showed a mean interventricular septal thickness (IVSD) of 18 ± 4.4 mm, a mean left ventricular ejection fraction (LVEF) of 63 ± 7.2%, and a mean LVOT Valsalva gradient of 64 ± 52 mmHg. Baseline therapy included beta-blockers (75.6%), calcium channel blockers (17.8%), and disopyramide (2%). 21.5% of patients did not require treatment escalation due to low LVOT gradients or minimal symptoms. 1.5% underwent transcatheter septal reduction therapy (SRT). Mavacamten was initiated in 77.7% of patients. A significant decrease in the LVOT Valsalva gradient was observed after 4 weeks (p0.001), with further reductions over 20 weeks. Simultaneously, NT-pro-BNP levels significantly decreased after 4 weeks (p0.001). At baseline, 50% of patients were classified as NYHA class III/IV, which improved to 11% after 20 weeks of treatment. 13 adverse events were reported within this period, including 3 cases of LVEF dropping below 50%, 9 instances of dizziness, and one case of syncope. Mavacamten was paused in all cases of LVEF reduction, with one case leading to permanent discontinuation. Conclusion These early findings from the German REDUCE-Registry demonstrate that myosin inhibitor therapy has been successfully integrated into clinical practice with three of four patients initiated with mavacamten. Consistent with results from approval trials, this treatment significantly reduced LVOT gradients and improved NYHA class in a substantial proportion of HOCM patients, affirming its safety and transformative potential in real-world settings.Table 1 Figure 1
Seuthe et al. (Sat,) reported a other. Mavacamten initiated in 77.7% HOCM patients reduced LVOT gradient and NT-pro-BNP significantly by 4 weeks, improving NYHA III/IV from 50% to 11% at 20 weeks.