Abstract：
Hypertrophic cardiomyopathy (HCM) is a phenotypically heterogeneous disease with a genetic basis and variable penetrance. The hallmarks of HCM include dynamic left ventricular outflow tract obstruction, typically caused by asymmetric septal hypertrophy. However, abnormal papillary muscle placement, abnormal mitral valve and subvalvular apparatus and apical hypertrophic forms have also been described. Typical medical treatment has been stagnant for decades, although there have been significant advances in surgical treatment of patients with obstructive HCM. Herein, we describe a new class of drugs targeting the specific pathophysiology of HCM.
Hypertrophic obstructive cardiomyopathy is a genetic condition that leads to increased heart muscle size. This increase in heart muscle can cause blockage of appropriate blood flow out of the heart. In such cases, current medications like β-blockers, calcium channel blockers and disopyramide can be used, but do not directly target the problem of increased heart muscle size. A new medication class, cardiac myosin inhibitors, decrease the squeezing power of the increased heart muscle to allow for more appropriate blood flow out of the heart. So far, trials have been conducted with mavacamten, with upcoming trials of aficamten (another novel cardiac myosin inhibitor). Recent trials with mavacamten have shown that this medication class has been beneficial for patients in whom other medications have failed. Some trials have also shown that by taking cardiac myosin inhibitors, patients have been able to avoid or delay surgery to correct this problem. Reassuringly, short-term data for this class of medications are positive. However, given that these medications are new, continued monitoring and research is being done to evaluate their long-term effect.
Hypertrophic obstructive cardiomyopathy is a genetic condition that leads to increased heart muscle size. This increase in heart muscle can cause blockage of appropriate blood flow out of the heart. In such cases, current medications like β-blockers, calcium channel blockers and disopyramide can be used, but do not directly target the problem of increased heart muscle size. A new medication class, cardiac myosin inhibitors, decrease the squeezing power of the increased heart muscle to allow for more appropriate blood flow out of the heart. So far, trials have been conducted with mavacamten, with upcoming trials of aficamten (another novel cardiac myosin inhibitor). Recent trials with mavacamten have shown that this medication class has been beneficial for patients in whom other medications have failed. Some trials have also shown that by taking cardiac myosin inhibitors, patients have been able to avoid or delay surgery to correct this problem. Reassuringly, short-term data for this class of medications are positive. However, given that these medications are new, continued monitoring and research is being done to evaluate their long-term effect.
摘要：
肥厚型心肌病（HCM）是一种表型异质性疾病，具有遗传基础和可变外显率。HCM的标志包括动态左心室流出道阻塞，通常由不对称间隔肥大引起。然而,异常的乳头状肌放置，还描述了异常的二尖瓣和瓣下器官以及心尖肥大形式。典型的医疗已经停滞了几十年，尽管在梗阻性HCM患者的手术治疗方面取得了重大进展。在本文中，我们描述了一类针对HCM特定病理生理学的新药。
肥厚型梗阻性心肌病是一种导致心肌大小增加的遗传性疾病。心肌的这种增加会导致适当的血液流出心脏的阻塞。在这种情况下,目前的药物如β受体阻滞剂，可以使用钙通道阻滞剂和丙吡胺，但不要直接针对心肌尺寸增加的问题。一种新的药物类别，心肌肌球蛋白抑制剂，减少增加的心肌的挤压力，以允许更适当的血液流出心脏。到目前为止,已经对Mavacampen进行了试验，即将进行的aficamten（另一种新型心脏肌球蛋白抑制剂）试验。最近使用mavacampen的试验表明，这种药物对其他药物失败的患者有益。一些试验还表明，通过服用心肌肌球蛋白抑制剂，患者已经能够避免或推迟手术来纠正这个问题。令人放心的是，此类药物的短期数据为阳性.然而,鉴于这些药物是新的，正在进行持续的监测和研究，以评估其长期效果。
肥厚型梗阻性心肌病是一种导致心肌大小增加的遗传性疾病。心肌的这种增加会导致适当的血液流出心脏的阻塞。在这种情况下,目前的药物如β受体阻滞剂，可以使用钙通道阻滞剂和丙吡胺，但不要直接针对心肌尺寸增加的问题。一种新的药物类别，心肌肌球蛋白抑制剂，减少增加的心肌的挤压力，以允许更适当的血液流出心脏。到目前为止,已经对Mavacampen进行了试验，即将进行的aficamten（另一种新型心脏肌球蛋白抑制剂）试验。最近使用mavacampen的试验表明，这种药物对其他药物失败的患者有益。一些试验还表明，通过服用心肌肌球蛋白抑制剂，患者已经能够避免或推迟手术来纠正这个问题。令人放心的是，此类药物的短期数据为阳性.然而,鉴于这些药物是新的，正在进行持续的监测和研究，以评估其长期效果。
