Cardiac myosin inhibitors: Efficacy, safety and future directions of aficamten in hypertrophic obstructive cardiomyopathy

心肌肌球蛋白抑制剂:阿菲卡坦治疗肥厚型梗阻性心肌病的疗效、安全性和未来方向

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Abstract

BACKGROUND: Hypertrophic obstructive cardiomyopathy (HOCM) is a genetic disorder that affects the cardiac myocytes leading to asymmetric hypertrophy of the left ventricle and obstruction of the left ventricular outflow tract (LVOT) with possible risk of sudden cardiac death in some high-risk patients. This paper aims to evaluate the efficacy, safety, and future prospects of aficamten in the management of HOCM and its potential to transform current standards of care. MAIN BODY: Over the years, there have been significant milestones in HOCM management, including pharmacological and surgical techniques, which have reduced the morbidity and mortality associated with this disease. However, HOCM remains a challenging disease to manage for healthcare providers due to the heterogeneous nature of its presentation and limitations to the conventional treatment medications including beta blockers and non-dihydropyridine calcium channel blockers. These traditional options often provide inadequate symptomatic relief and have significant side effects. Surgical options such as septal ablation have shown positive outcomes but are associated with procedural risks. Therefore, recent advancements have led to the development of novel agents such as cardiac myosin inhibitors (CMIs). CONCLUSION: Aficamten is a new second-generation cardiac myosin inhibitor (CMI) that has shown promising results from clinical trials. With reports of reduced LVOT obstruction and improvement in heart failure symptoms, these findings indicate potential improvement in the quality of life of patients with HOCM.

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