Current cardiology reports · 2025
Mavacamten in the Treatment of Obstructive Hypertrophic Cardiomyopathy: from Pathophysiology To Real World Data.
СтатьяОбзорНазвание и аннотация: Перевод в очереди
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Panichella Giorgia, Garofalo Manuel, Ragagnin Maddalena, Fanizzi Angela Ilaria, Zampieri Mattia, Del Franco Annamaria, Cappelli Francesco, Pieroni Maurizio, Olivotto Iacopo
Аннотация
PURPOSE OF REVIEW: This review aims to provide a comprehensive overview of mavacamten in the management of obstructive hypertrophic cardiomyopathy (oHCM), from its molecular mechanism of action to clinical trial outcomes and real-world application. It explores the efficacy, safety, and practical considerations of mavacamten use, while highlighting evolving indications and future directions.
RECENT FINDINGS: Randomized trials have shown that mavacamten significantly reduces left ventricular outflow tract gradients, improves symptoms and exercise capacity, and induces structural reverse remodeling with a favorable safety profile. Real-world data confirm these benefits in broader patient populations. Pharmacogenetic variability, titration protocols, and cost-effectiveness analyses have further refined its clinical use. Mavacamten represents a paradigm shift in oHCM treatment by targeting disease at its sarcomeric origin. Its integration into routine care is expanding, supported by real-world evidence. Ongoing studies will clarify its role in non-obstructive HCM, heart failure with preserved ejection fraction, pediatric populations, and early-stage disease.
RECENT FINDINGS: Randomized trials have shown that mavacamten significantly reduces left ventricular outflow tract gradients, improves symptoms and exercise capacity, and induces structural reverse remodeling with a favorable safety profile. Real-world data confirm these benefits in broader patient populations. Pharmacogenetic variability, titration protocols, and cost-effectiveness analyses have further refined its clinical use. Mavacamten represents a paradigm shift in oHCM treatment by targeting disease at its sarcomeric origin. Its integration into routine care is expanding, supported by real-world evidence. Ongoing studies will clarify its role in non-obstructive HCM, heart failure with preserved ejection fraction, pediatric populations, and early-stage disease.