Key result
Real-world mavacamten linked to ~63 mmHg drop in peak LVOT gradients over 12 weeks.
Why the study?
Clinical experience and manageability of symptomatic oHCM patients treated with mavacamten in a real-world setting were still lacking.
Does mavacamten improve quality of life, serum biomarkers, and LVOT gradients in symptomatic oHCM patients in a real-world setting?
Observational (n=15)
No
Does mavacamten improve quality of life, serum biomarkers, and LVOT gradients in symptomatic oHCM patients in a real-world setting?
p-value: p=0.0002
Real-world initiation of mavacamten in oHCM patients over 12 weeks confirms clinical trial findings of improved symptoms, biomarkers, and LVOT gradients, though careful monitoring for transient LVEF drop is required.
Requires LVEF monitoring during mavacamten titration in oHCM; extends trial data observationally but leaves open randomized durability evidence.
INTRODUCTION: Lately, mavacamten emerged as a new therapeutic option for symptomatic patients with obstructive hypertrophic cardiomyopathy (oHCM). Clinical trials revealed reduction of serum biomarkers, and left ventricular outflow tract (LVOT) obstruction, as well as an improvement in clinical symptoms and exercise capacity. Nevertheless, clinical experience and manageability of patients in a real-world setting is still lacking. MATERIAL AND METHODS: 22 patients with symptomatic oHCM (54.5% male, age 58.5 ± 16.2 years) and elevated LVOT gradients were started on mavacamten between March 2023 and June 2024. All patients were New York Heart Association (NYHA) class II or higher. Seven patients were excluded from primary analysis due to comedication with Angiotensin-converting-enzyme-inhibitors or Angiotensin-II receptor blockers. Cardiac imaging, laboratory work-up and clinical evaluation were assessed at three visits during the 12 weeks initiation phase; Dosing of mavacamten was adjusted according to manufacturer's recommendations. RESULTS: At 12 weeks, the majority of patients described a significant improvement of their quality of life. Work-up at 12 weeks revealed a significant reduction of serum biomarkers and LVOT gradients. In four patients, mavacamten needed to be temporarily paused due to clinical complaints or transient left ventricular ejection fraction deterioration below 50% with subsequent full recovery. CONCLUSION: We provide first insights into the usage of mavacamten in oHCM patients during the titration period in a real-world setting. Clinical findings are in line with previous clinical trials. In accordance with current recommendations, we highlight the need for standardized follow-up of patients on mavacamten treatment.
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Becker et al. (2024) conducted an observational in Obstructive hypertrophic cardiomyopathy (oHCM) (n=15). Mavacamten vs. Baseline was evaluated on Mean change in peak LVOT gradient at 12 weeks (95% CI 31.1; 95.7, p=0.0002). In a real-world cohort of patients with symptomatic obstructive hypertrophic cardiomyopathy, mavacamten significantly reduced mean peak LVOT gradients by 63.4 mmHg over a 12-week titration period.
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