冠状动脉肌浆网钙 ATP 酶基因治疗对射血分数降低的晚期心力衰竭患者：一项前瞻性队列研究。
Intracoronary Sarcoplasmic Reticulum Calcium-ATPase Gene Therapy in Advanced Heart Failure Patients with reduced Ejection Fraction: A Prospective Cohort Study.
机构信息
Cardio-Pulmonary Rehabilitation Inpatient Area, Second Rehabilitation Hospital of Shanghai, Shanghai 200431, China.
出版信息
Clinics (Sao Paulo). 2020;75:e1530. doi: 10.6061/clinics/2020/e1530. Epub 2020 Mar 13.
OBJECTIVE
Heart failure is a progressive and debilitating disease. Intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy may improve the function of cardiac muscle cells. This study aimed to test the hypothesis that intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy can improve outcomes and reduce the number of recurrent and terminal events in advanced heart failure patients with reduced ejection fraction.
METHODS
A total of 768 heart failure patients with reduced ejection fraction and New York Heart Association classification II to IV were included in this prospective cohort study. Patients either underwent intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy (CA group, n=384) or received oral placebo (PA group; n=384). Data regarding recurrent and terminal event(s), treatment-emergent adverse effects, and outcome measures were collected and analyzed.
RESULTS
After a follow-up period of 18 months, intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy reduced the number of hospital admissions (p=0.001), ambulatory treatments (p=0.0004), and deaths (p=0.024). Additionally, intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy improved the left ventricular ejection fraction (p<0.0001) and Kansas City Cardiomyopathy Questionnaire score (p<0.0001). The number of recurrent and terminal events/patients were higher in the PA group than in the CA group after the follow-up period of 18 months (p=0.015). The effect of the intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy was independent of the confounding variables. No new arrhythmias were reported in the CA group.
CONCLUSIONS
Intracoronary sarcoplasmic reticulum calcium-ATPase gene therapy reduces the number of recurrent and terminal events and improves the clinical course of advanced heart failure patients with reduced ejection fraction.
目的
心力衰竭是一种进行性和使人虚弱的疾病。肌浆网钙 ATP 酶基因治疗可能改善心肌细胞的功能。本研究旨在检验这样一个假设，即冠状动脉肌浆网钙 ATP 酶基因治疗可以改善射血分数降低的晚期心力衰竭患者的预后，并减少复发和终末事件的发生。
方法
本前瞻性队列研究共纳入 768 例射血分数降低且纽约心脏协会分级 II 至 IV 级的心力衰竭患者。患者接受冠状动脉肌浆网钙 ATP 酶基因治疗（CA 组，n=384）或口服安慰剂（PA 组；n=384）。收集并分析复发和终末事件、治疗中出现的不良事件和结局指标的数据。
结果
在 18 个月的随访期后，冠状动脉肌浆网钙 ATP 酶基因治疗减少了住院次数（p=0.001）、门诊治疗次数（p=0.0004）和死亡人数（p=0.024）。此外，冠状动脉肌浆网钙 ATP 酶基因治疗改善了左心室射血分数（p<0.0001）和堪萨斯城心肌病问卷评分（p<0.0001）。在 18 个月的随访期后，PA 组的复发和终末事件/患者数量高于 CA 组（p=0.015）。冠状动脉肌浆网钙 ATP 酶基因治疗的效果与混杂变量无关。CA 组未报告新发心律失常。
结论
冠状动脉肌浆网钙 ATP 酶基因治疗减少了复发和终末事件的发生，并改善了射血分数降低的晚期心力衰竭患者的临床病程。
Zotero 插件