Ogieuhi Ikponmwosa Jude, Ajekiigbe Victor Oluwatomiwa, Adeyemi Boluwaduro Abasiekem, Nwatamole Bright, Ayodeji Komolafe Babajide, Ugiomoh Oshomoh Mark-Anthony, Odeniyi Temiloluwa Adebayo, Akingbola Adewunmi, Iyawe Efosa Peace, Oladejo Olabode Olawale, Lawal Motunrayo Oluwatoyosi, Woldehana Nathnael Abera, Bakare Ifeoluwa Sandra, Patience Adejumo Temilade, Okoro Grace Chinenye
Siberian State Medical University, Tomsk, Russian Federation.
Ladoke Akintola University of Technology, Ogbomoso, Nigeria.
Egypt Heart J. 2025 Jun 13;77(1):61. doi: 10.1186/s43044-025-00652-0.
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.
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).
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.
肥厚型梗阻性心肌病（HOCM）是一种遗传性疾病，会影响心肌细胞，导致左心室不对称肥厚以及左心室流出道（LVOT）梗阻，部分高危患者可能有心脏性猝死风险。本文旨在评估阿非卡坦治疗HOCM的疗效、安全性及未来前景，以及其改变当前治疗标准的潜力。
多年来，HOCM的治疗取得了重大进展，包括药物和手术技术，这些都降低了与该疾病相关的发病率和死亡率。然而，由于其临床表现的异质性以及传统治疗药物（包括β受体阻滞剂和非二氢吡啶类钙通道阻滞剂）的局限性，HOCM对医疗服务提供者来说仍然是一种具有挑战性的疾病。这些传统选择往往无法充分缓解症状，且有明显的副作用。诸如室间隔消融等手术选择已显示出积极效果，但与手术风险相关。因此，最近的进展促使了新型药物如心肌肌球蛋白抑制剂（CMIs）的研发。
阿非卡坦是一种新型第二代心肌肌球蛋白抑制剂（CMI），临床试验已显示出有前景的结果。随着左心室流出道梗阻减轻和心力衰竭症状改善的报道，这些发现表明HOCM患者的生活质量可能得到改善。
