医学
肥厚性心肌病
临床试验
心脏病学
内科学
心源性猝死
心室流出道梗阻
随机对照试验
猝死
心力衰竭
重症监护医学
心肌病
疾病
心脏病
作者
Jesse Cole,Samantha F. Benvie,Marci DeLosSantos
标识
DOI:10.1016/j.clinthera.2024.02.007
摘要
Hypertrophic cardiomyopathy (HCM) is an under-recognized genetic cardiac disorder affecting the muscles and contractility of the heart, which in turn can result in heart failure symptoms, arrhythmia, and sudden cardiac death. Previously, pharmacotherapy options for HCM were not disease-specific, often poorly tolerated, and overall inadequate for optimal management. This narrative review discusses the pharmacology of the novel drug mavacamten, the clinical trials supporting its use, and considerations for its use in clinical practice.PubMed and ClinicalTrials.gov were searched for the key words mavacamten and Camzyos to identify currently active clinical trials and clinical trials published between January 2015 and March 2023. Data from EXPLORER-HCM were included, as EXPLORER-HCM led to approval by the US Food and Drug Administration of the use of mavacamten, along with data from VALOR-HCM, which provided additional evidence for use. Publications that were not randomized, controlled trials were not included in this review.The findings from this review suggest that mavacamten is an effective treatment for patients with persistently symptomatic obstructive HCM and may decrease the need for septal reduction therapy. Mavacamten use was associated with improved exercise capacity, left ventricular outflow tract obstruction, and New York Heart Association functional class, and with a decreased frequency of septal reduction therapy.HCM is associated with significant morbidity and mortality, independent of other disease states. Mavacamten is a novel treatment option for patients with HCM and offers an additional option for patients with persistent symptoms who previously had limited treatment options. The use of mavacamten in patients with obstructive HCM may improve exercise capacity, and decrease symptoms and the need for septal reduction therapy. There is potential for mavacamten to be indicated for use in patients with nonobstructive HCM in the future, pending findings from Phase III trials in this population.
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