EFFICACY AND SAFETY OF MAVACAMTEN IN HYPERTROPHIC CARDIOMYOPATHY – A SYSTEMATIC REVIEW OF CURRENT CLINICAL STUDIES
DOI:
https://doi.org/10.31435/ijitss.2(50).2026.5881Keywords:
Mavacamten; Cardiac Myosin Inhibitor; Hypertrophic Cardiomyopathy; LVOT ObstructionAbstract
Introduction: Hypertrophic cardiomyopathy (HCM) is an inherited condition characterized by sarcomeric hypercontractility, leading to dynamic left ventricular outflow tract (LVOT) obstruction in approximately 70% of patients. Mavacamten, a first-in-class selective cardiac myosin inhibitor, addresses the underlying molecular cause by reducing excessive actin-myosin cross-bridge formation.
Aim: To evaluate the clinical efficacy and safety of mavacamten in symptomatic obstructive HCM (oHCM) using data from randomized trials and real-world studies.
Methods: A systematic search of PubMed, Embase, and Cochrane Library identified 110 publications. Following predefined criteria, 44 studies were included in the final analysis, including pivotal phase III trials (EXPLORER-HCM, VALOR-HCM) and long-term extensions.
Results: Mavacamten demonstrated a significant reduction in LVOT gradients (mean reduction of ~47 mmHg in EXPLORER-HCM) and improved quality of life, with KCCQ-CSS scores increasing by an average of 14.2 points. Between 60% and 80% of patients achieved ≥1 NYHA functional class improvement. In the VALOR-HCM trial, approximately 90% of patients avoided invasive septal reduction therapy (SRT) through 128 weeks. The primary safety concern was a transient reduction in LVEF (<50%) occurring in 5–14% of patients, which was typically reversible with dose titration.
Conclusion: Mavacamten is a transformative non-invasive therapy that shifts oHCM management from palliative care to mechanism-targeted treatment. While highly effective in improving hemodynamics and functional capacity, rigorous echocardiographic monitoring remains mandatory to ensure safety regarding systolic function.
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Copyright (c) 2026 Wiktoria Szkatuła, Marcin Schulz, Agnieszka Morawa, Dawid Studziński, Karolina Osińska, Michał Kulczak, Anna Kołcz
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