Key result
Mavacamten is associated with a ~59 mmHg reduction in resting peak LVOT gradient in oHCM.
Why the study?
Real-world data on the effectiveness and safety of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy were scarce.
Does mavacamten improve left ventricular outflow tract gradients, cardiac biomarkers, and NYHA functional class safely in a real-world cohort of patients with symptomatic obstructive hypertrophic cardiomyopathy?
Observational (n=133)
Yes
Does mavacamten improve left ventricular outflow tract gradients, cardiac biomarkers, and NYHA functional class safely in a real-world cohort of patients with symptomatic obstructive hypertrophic cardiomyopathy?
This multicentre real-world registry confirms the findings of pivotal clinical trials, demonstrating that mavacamten substantially reduces LVOT gradients, cardiac biomarkers, and heart failure symptoms with a manageable safety profile in patients with obstructive hypertrophic cardiomyopathy.
Supports real-world mavacamten use in symptomatic oHCM; extends pivotal trials yet leaves practice change to future RCTs.
Background/Objectives Mavacamten is a selective cardiac myosin inhibitor indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Given the scarcity of real-world data, we aimed to investigate the effectiveness and safety of mavacamten in patients enrolled in the MARVEL.HU (MAvacamten Real-world eVidencE during short- and Long-term follow-up in hypertrophic cardiomyopathy in HUngary) multicentre registry. Patients and Methods One-hundred and thirty-three patients with oHCM [71 males (53%), average age: 56±13 years] receiving mavacamten treatment were followed. Titration and follow-up followed the standard instructions for use of mavacamten. The follow-up was an average of 239±158 days [median 226 days (146-302)]; the number of patients completing the week 4 (W4), W12, W24, and W48 visits was 125 (94%), 120 (90%), 105 (79%), and 31 (23%), respectively. Results The mean resting peak left ventricular outflow tract gradient (LVOTG) was 78 mmHg at baseline (W0) and decreased by a mean of 34 mmHg (95% CI: 41 to 27) at W4, 44 mmHg (95% CI: 52 to 37) at W12, and 59 mmHg (95% CI: 68 to 49) at W24, reaching 18 mmHg in patients completing W48 visits. The LVOTG provoked by the Valsalva manoeuvre of 110 mmHg at W0 decreased by 39 mmHg (95% CI: 47 to 31), 52 mmHg (95% CI: 62 to 42), and 74 mmHg (95% CI: 84 to 63) at W4, W12 and W24, respectively; falling to 28 mmHg at W48. Median NTproBNP level of 945 pg/ml at baseline decreased by 696 pg/ml (95% CI: 950 to 495), 915 pg/ml (95% CI: 1265 to 640), and 1175 pg/ml (95% CI: 1613 to 852), at W4, W12 and W24, respectively; showing further decrease to 159 pg/ml at W48. Troponin T levels also showed substantial decrease by 12,5 ng/l (95% CI: 21,5 to 7), from 18 to 9,5 ng/l at W48. NYHA functional class improved ≥1 class in 23%, 53%, 71% and 86% of patients at W4, W12, W24 and W48, respectively, and ≥2 class in 3%, 9%, 19% and 31% of patients at the same time points. Left ventricular ejection fraction (LVEF) didn’t change significantly, the decrease of LVEF was 2,9% (95% CI: 5,6 to 0,3) at W48. During the study, 20 patients (15%) experienced an LVEF <55%, while 5 patients (3.8%) fell to ≤50%(one each at W4, W20, W48 and W56, two at W36). All five patients recovered their LVEF after temporary drug interruption and successfully restarted treatment. There was one death (one case of sudden cardiac death) during follow-up, which was deemed unrelated to the treatment. Conclusions These real-world findings corroborate the results of pivotal clinical trials regarding the effectiveness and safety of mavacamten in patients with oHCM.
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Sepp et al. (2026) conducted an observational in Symptomatic obstructive hypertrophic cardiomyopathy (n=133). Mavacamten was evaluated on Decrease in mean resting peak left ventricular outflow tract gradient (LVOTG) from baseline to week 24 (MD -59 mmHg, 95% CI -68 to -49). Mavacamten reduced mean resting peak LVOT gradient by 59 mmHg (95% CI: 49-68) at week 24 and improved NYHA class by ≥1 in 86% of oHCM patients at week 48.
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