Key result
Mavacamten is linked to ~9-fold higher odds of ≥30 mmHg LVOT gradient reduction vs conventional therapy.
Why the study?
Many patients with symptomatic HOCM do not respond to conventional agents, and the dose-time response of mavacamten in routine practice remains unclear.
Does clinically guided mavacamten therapy reduce resting LVOT gradient and BNP in symptomatic HOCM patients compared to conventional pharmacotherapy?
Cohort (n=95)
No
Does clinically guided mavacamten therapy reduce resting LVOT gradient and BNP in symptomatic HOCM patients compared to conventional pharmacotherapy?
In symptomatic HOCM, clinically guided mavacamten therapy significantly increases the likelihood of achieving a ≥30 mmHg reduction in resting LVOT gradient compared to conventional pharmacotherapy, without compromising LVEF.
Mavacamten was associated with echocardiographic improvements in HOCM; leaves open randomized confirmation versus conventional therapy.
Background Hypertrophic obstructive cardiomyopathy (HOCM) causes dynamic left ventricular outflow tract (LVOT) obstruction and disabling symptoms; many patients do not respond to conventional agents. Mavacamten, a selective cardiac myosin inhibitor, improves gradients, but the dose–time response in routine practice remains unclear. Aims To evaluate longitudinal echocardiographic, biomarker, and safety changes associated with clinically guided mavacamten therapy compared with conventional pharmacotherapy in symptomatic HOCM. Methods In this single‐center prospective comparative cohort study, 95 symptomatic HOCM patients were included, of whom 48 received mavacamten and 47 received conventional pharmacotherapy. Mavacamten was initiated at 2.5 mg daily and titrated (2.5–10 mg) based on resting LVOT gradient (LVOTG) and ejection fraction (LVEF). Follow‐up assessments were performed at baseline and at scheduled visits at 3, 6, and 9 months. Longitudinal echocardiographic trajectories were analyzed using observed mean changes with 95% confidence intervals (CIs), and baseline imbalance was assessed using inverse probability weighting. The predefined LVOTG response endpoint was defined as a reduction in resting LVOTG of ≥ 30 mmHg from baseline at the last available follow‐up. Results Mavacamten was associated with a downward trajectory in resting LVOTG during follow‐up, whereas the control group remained relatively stable. BNP decreased significantly in the mavacamten group, whereas no significant paired change was observed in the control group. The predefined LVOTG response endpoint was achieved by 9 of 48 patients (18.8%) in the mavacamten group and 1 of 42 patients (2.4%) in the control group (Fisher exact p = 0.0175; OR = 9.46, 95% CI 1.14–78.20). No patient experienced LVEF < 50%. Conclusions In symptomatic HOCM, clinically guided mavacamten therapy was associated with reductions in resting LVOT gradient and BNP, while LVEF remained preserved during short‐term follow‐up. These findings support individualized dose titration with echocardiographic monitoring, while dose‐stratified and response analyses should be interpreted cautiously because of limited subgroup sizes.
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Cao et al. (2026) conducted a cohort in Symptomatic hypertrophic obstructive cardiomyopathy (HOCM) (n=95). Mavacamten vs. Conventional pharmacotherapy was evaluated on Reduction in resting LVOTG of ≥ 30 mmHg from baseline at the last available follow-up (OR 9.46, 95% CI 1.14-78.20, p=0.0175). In symptomatic HOCM, mavacamten was associated with a higher rate of ≥30 mmHg reduction in resting LVOT gradient compared to conventional therapy (18.8% vs 2.4%; OR 9.46, 95% CI 1.14-78.20).
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