HCMBeat.com, Los Angeles, CA, USA.
Patient author.
Future Cardiol. 2021 Oct;17(7):1269-1275. doi: 10.2217/fca-2021-0044. Epub 2021 May 21.
Mavacamten is an investigational therapy for the treatment of hypertrophic cardiomyopathy (HCM), a condition where the heart muscle wall thickens, becomes stiff, and makes it harder for the heart to pump blood. In obstructive HCM (sometimes referred to as oHCM or HOCM), the thickened muscle also blocks blood flow from the heart. The EXPLORER-HCM trial compared mavacamten to placebo (a pill with no medicine/active substances) in symptomatic people with obstructive HCM who had exercise limitations and suffered from shortness of breath, tiredness, palpitations, and chest pain. The study showed that mavacamten reduced the obstruction that restricts blood flow and improved people's symptoms, well-being, and ability to participate in daily activities. Side effects, such as irregular heartbeat, palpitations, rapid heartbeat, and heart failure, were similar for people who received mavacamten or placebo. Clinical Trial Registration: NCT03470545 (ClinicalTrials.gov).
马卡塞顿是一种治疗肥厚型心肌病（HCM）的研究性疗法，HCM 是一种心肌壁变厚、变硬，使心脏更难泵血的疾病。在梗阻性 HCM（有时称为 oHCM 或 HOCM）中，增厚的肌肉也会阻止血液从心脏流出。EXPLORER-HCM 试验比较了马卡塞顿与安慰剂（一种不含药物/活性物质的药丸）在有运动受限且有呼吸困难、疲劳、心悸和胸痛等症状的梗阻性 HCM 患者中的疗效。研究表明，马卡塞顿减少了限制血液流动的阻塞，改善了患者的症状、整体健康状况和参与日常活动的能力。接受马卡塞顿或安慰剂的患者的副作用（如心跳不规则、心悸、心跳过快和心力衰竭）相似。临床试验注册：NCT03470545（ClinicalTrials.gov）。
