ORIGINAL RESEARCH article
Front. Cardiovasc. Med.
Sec. Heart Failure and Transplantation
This article is part of the Research TopicTransforming Care in Heart Failure and Cardiomyopathies: Emerging Insights and TreatmentsView all 12 articles
Hemodynamic and symptomatic response in hypertrophic obstructive cardiomyopathy patients on myosin inhibitor therapy
Provisionally accepted- 1Cardiology, University Hospital of Cologne, Cologne, Germany
- 2Radiology, University Hospital of Cologne, Cologne, Germany
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Aims To provide real-world data on the symptomatic and hemodynamic response of the myosin inhibitor mavacamten in patients with hypertrophic obstructive cardiomyopathy. Methods Patients with HOCM up-titrated to their final mavacamten dose were included. The final dose was defined as (i) 15 mg daily (or 5 mg for poor CYP2C19 metabolizers), (ii) a dose achieving a complete hemodynamic response (LVOT gradient < 30 mmHg), or (iii) a lower dose limited by adverse effects. Final evaluation was performed 12 weeks after reaching the final dose. Symptomatic response was defined as ≥1 NYHA class improvement, and incomplete hemodynamic response as residual LVOT gradient ≥ 30 mmHg. Results 40 patients (56 ± 12 years, 78% male) were included. The LVOT gradient (rest: -25 ± 32mmHg, p <0.001, Valsalva: -73 ± 56mmHg, p <0.001) and NT-proBNP levels (-785 ± 1,122ng/l, p <0.001) significantly decreased during a mean follow up of 184 ± 83 days. 78% had a symptomatic response and 93% were complete hemodynamic responders. Patients with no improvement in NYHA class had a lower e´ lat. (9 ± 3cm/s vs. 6 ± 2cm/s, p=0.034) and less often baseline therapy with beta-blockers. Patients with incomplete hemodynamic response had a significantly higher baseline septum thickness (26.3 ± 4.9mm vs 19.1 ± 3.7mm, p=0.007) and higher LV-mass index (205 ± 63mL/m² vs. 139 ± 31mL/m², p=0.038). Absolute reduction of LVOT gradients was similar in patients with and without clinical or hemodynamic response. Conclusion Clinical and hemodynamic response to mavacamten was high in this real-world cohort and comparable to pivotal trial results. Incomplete response might be related to more severe baseline disease, which needs further study.
Keywords: Hypertrophic obstructive cardiomyopathy, Myosin-Inhibitor, Mavacamten, hemodynamic response, Symptomatic response
Received: 02 Jun 2025; Accepted: 14 Nov 2025.
Copyright: © 2025 Seuthe, Feidakis, Nies, Brüwer, Kaya, Pennig, Ten Freyhaus, Baldus and Pfister. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Katharina Seuthe, katharina.seuthe@uk-koeln.de
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