Abstract：
UNASSIGNED: Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic heart disease with an estimated prevalence in the general population of 0.2% to 0.6%. Clinically, HCM can range from no symptoms to severe symptoms such as heart failure or sudden cardiac death. Currently, the management of HCM involves lifestyle modifications, familial screening, genetic counseling, pharmacotherapy to manage symptoms, sudden cardiac death risk assessment, septal reduction therapy, and heart transplantation for specific patients. Multicenter randomized controlled trials have only recently explored the potential of cardiac myosin inhibitors (CMIs) such as mavacamten as a directed pharmacological approach for managing HCM.
UNASSIGNED: We will assess the existing medical treatments for HCM: beta-blockers, calcium channel blockers, disopyramide, and different CMIs. We will also discuss future HCM pharmacotherapy guidelines and underline this patient population\'s unfulfilled needs.
UNASSIGNED: Mavacamten is the first-in-class CMI approved by the FDA to target HCM pathophysiology specifically. Mavacamten should be incorporated into the standard therapy for oHCM in case of symptom persistence despite using maximally tolerated beta blockers and/or calcium channel blockers. Potential drug-drug interactions should be assessed before initiating this drug. More studies are needed on the use of CMIs in patients with kidney and/or liver failure and pregnant/breastfeeding patients.
UNASSIGNED: We will assess the existing medical treatments for HCM: beta-blockers, calcium channel blockers, disopyramide, and different CMIs. We will also discuss future HCM pharmacotherapy guidelines and underline this patient population\'s unfulfilled needs.
UNASSIGNED: Mavacamten is the first-in-class CMI approved by the FDA to target HCM pathophysiology specifically. Mavacamten should be incorporated into the standard therapy for oHCM in case of symptom persistence despite using maximally tolerated beta blockers and/or calcium channel blockers. Potential drug-drug interactions should be assessed before initiating this drug. More studies are needed on the use of CMIs in patients with kidney and/or liver failure and pregnant/breastfeeding patients.
摘要：
■肥厚型心肌病（HCM）是一种异质性遗传性心脏病，在普通人群中的患病率估计为0.2％至0.6％。临床上,HCM可以从无症状到严重症状，如心力衰竭或心脏猝死。目前,HCM的管理涉及生活方式的改变，家族筛查,遗传咨询,药物治疗来控制症状，心源性猝死风险评估,中隔缩小治疗,以及针对特定患者的心脏移植。多中心随机对照试验直到最近才探索了心脏肌球蛋白抑制剂（CMI）（例如mavacamten）作为管理HCM的定向药理学方法的潜力。
■我们将评估HCM的现有药物治疗方法：β受体阻滞剂，钙通道阻滞剂，丙吡胺,不同的CMI。我们还将讨论未来的HCM药物治疗指南，并强调该患者人群的未满足需求。
■Mavacampen是FDA批准的一流CMI，专门针对HCM病理生理学。尽管使用了最大耐受的β受体阻滞剂和/或钙通道阻滞剂，但如果症状持续存在，则应将Mavacamten纳入oHCM的标准治疗。在开始使用该药物之前，应评估潜在的药物-药物相互作用。需要对肾脏和/或肝脏衰竭患者以及怀孕/母乳喂养患者使用CMI进行更多研究。
■我们将评估HCM的现有药物治疗方法：β受体阻滞剂，钙通道阻滞剂，丙吡胺,不同的CMI。我们还将讨论未来的HCM药物治疗指南，并强调该患者人群的未满足需求。
■Mavacampen是FDA批准的一流CMI，专门针对HCM病理生理学。尽管使用了最大耐受的β受体阻滞剂和/或钙通道阻滞剂，但如果症状持续存在，则应将Mavacamten纳入oHCM的标准治疗。在开始使用该药物之前，应评估潜在的药物-药物相互作用。需要对肾脏和/或肝脏衰竭患者以及怀孕/母乳喂养患者使用CMI进行更多研究。
