Vanderbilt Center for Arrhythmia Research and Therapeutics, Division of Clinical Pharmacology, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
J Physiol. 2020 Jul;598(14):2817-2834. doi: 10.1113/JP276757. Epub 2020 Apr 27.
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a stress-induced cardiac channelopathy that has a high mortality in untreated patients. Our understanding has grown tremendously since CPVT was first described as a clinical syndrome in 1995. It is now established that the deadly arrhythmias are caused by unregulated 'pathological' calcium release from the sarcoplasmic reticulum (SR), the major calcium storage organelle in striated muscle. Important questions remain regarding the molecular mechanisms that are responsible for the pathological calcium release, regarding the tissue origin of the arrhythmic beats that initiate ventricular tachycardia, and regarding optimal therapeutic approaches. At present, mutations in six genes involved in SR calcium release have been identified as the genetic cause of CPVT: RYR2 (encoding ryanodine receptor calcium release channel), CASQ2 (encoding cardiac calsequestrin), TRDN (encoding triadin), CALM1, CALM2 and CALM3 (encoding identical calmodulin protein). Here, we review each CPVT subtype and how CPVT mutations alter protein function, RyR2 calcium release channel regulation, and cellular calcium handling. We then discuss research and hypotheses surrounding the tissue mechanisms underlying CPVT, such as the pathophysiological role of sinus node dysfunction in CPVT, and whether the arrhythmogenic beats originate from the conduction system or the ventricular working myocardium. Finally, we review the treatments that are available for patients with CPVT, their efficacy, and how therapy could be improved in the future.
儿茶酚胺多形性室性心动过速(CPVT)是一种应激诱导的心脏通道病,如果未经治疗,患者死亡率很高。自 1995 年首次描述为临床综合征以来,我们对 CPVT 的认识已经有了很大的提高。现在已经确定,致命性心律失常是由肌浆网(SR)中不受调节的“病理性”钙释放引起的,SR 是横纹肌中主要的钙储存细胞器。对于导致病理性钙释放的分子机制、引发室性心动过速的心律失常起源的组织来源以及最佳治疗方法,仍存在重要问题。目前,已经确定 6 个参与 SR 钙释放的基因中的突变是 CPVT 的遗传原因:RYR2(编码肌质网钙释放通道)、CASQ2(编码心脏钙网蛋白)、TRDN(编码三联蛋白)、CALM1、CALM2 和 CALM3(编码相同的钙调蛋白蛋白)。在这里,我们回顾了每种 CPVT 亚型以及 CPVT 突变如何改变蛋白功能、RYR2 钙释放通道调节和细胞钙处理。然后,我们讨论了 CPVT 潜在组织机制的研究和假设,例如窦房结功能障碍在 CPVT 中的病理生理作用,以及心律失常起源于传导系统还是心室工作心肌。最后,我们回顾了 CPVT 患者可用的治疗方法、其疗效以及未来如何改进治疗方法。