PDF(Sci-hub)
Abstract：
Hypertrophic cardiomyopathy (HCM) is the most common monogenic cardiac disease with a highly variable phenotypic expression, ranging from asymptomatic to drug refractory heart failure (HF) presentation. Pharmacological therapy is the first line of treatment, but options are currently limited to nonspecific medication like betablockers or calcium channel inhibitors, with frequent suboptimal results. While being the gold standard practice for the management of drug refractory HCM patients, septal reduction therapy (SRT) remains an invasive procedure with associated surgical risks and it requires the expertise of the operating centre, thus limiting its accessibility. It is therefore with high interest that researchers look for pharmacological alternatives that could provide higher rates of success. With new data gathering these past years as well as the development of a new drug class showing promising results, this review provides an up-to-date focused synthesis of existing medical treatment options and future directions for HCM pharmacological treatment.
摘要：
肥厚型心肌病（HCM）是最常见的单基因心脏病，具有高度可变的表型表达，从无症状到药物难治性心力衰竭（HF）。药物治疗是一线治疗,但目前的选择仅限于非特异性药物，如β受体阻滞剂或钙通道抑制剂，频繁的次优结果。虽然是药物难治性HCM患者管理的黄金标准做法，间隔减少治疗（SRT）仍然是具有相关手术风险的侵入性程序，需要手术中心的专业知识。从而限制了它的可达性。因此，研究人员非常感兴趣地寻找可以提供更高成功率的药理学替代品。随着过去几年收集的新数据以及新药类别的开发显示出有希望的结果，这篇综述提供了HCM药物治疗的现有药物治疗方案和未来方向的最新重点综述.
