Randomized, double-blind, phase I/II study of intravenous allogeneic mesenchymal stromal cells in acute myocardial infarction
Randomized, double-blind, phase I/II study of intravenous allogeneic mesenchymal stromal cells in acute myocardial infarction
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DOI：
10.1016/j.jcyt.2014.10.009
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发表时间：
2015-03-01
期刊：
影响因子：
4.5
通讯作者：
Gupta, Pawan Kumar
中科院分区：
文献类型：
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作者：
Chullikana, Anoop;Sen Majumdar, Anish;Gupta, Pawan Kumar
背景目标。细胞治疗作为一种探索性心血管治疗方法具有广阔的应用前景。我们最近开发了一种名为Stempeucel(骨髓来源的同种异体间充质基质细胞)的研究新药，用于治疗ST段抬高的急性心肌梗死(AMI)患者。我们进行了一项I/II期随机、双盲、单次给药的研究，以评估静脉注射Stempeucel与安慰剂(多电解质注射)的安全性和有效性。方法：研究方法。将20例接受经皮冠状动脉介入治疗的急性心肌梗死患者按1：1的比例随机分成两组，分别给予丹皮酚静脉注射和安慰剂治疗，并进行2年的随访。结果。在Stempeucel组和安慰剂组中，观察到的紧急治疗不良事件的数量分别为18和21。根据调查人员和独立数据安全监测委员会的说法，所有不良事件都与Stempeucel无关。Stempeucel组未发生严重不良事件，安慰剂组有3例严重不良事件，其中1例死亡。结果。超声心动图测定的射血分数在6个月时Stempeucel组(43.06%至47.80%)和安慰剂组(43.44%至45.33%)均有改善(P=0.26)。在6个月时，使用单光子发射断层扫描测量的血流灌注分数和使用磁共振成像测量的脑梗塞体积显示两组之间没有显著差异。结论。这项研究表明，在急性心肌梗死患者经皮冠状动脉介入治疗2天后静脉给药时，Stempeucel是安全和耐受性良好的。最佳给药剂量和给药途径需要在更大的临床试验(http://clinicaltrials.gov/show/NCT00883727).)中进一步评估
Background aims. Cell therapy is promising as an exploratory cardiovascular therapy. We have recently developed an investigational new drug named Stempeucel (bone marrow-derived allogeneic mesenchymal stromal cells) for patients with acute myocardial infarction (AMI) with ST-segment elevation. A phase I/II randomized, double-blind, single-dose study was conducted to assess the safety and efficacy of intravenous administration of Stempeucel versus placebo (multiple electrolytes injection). Methods. Twenty patients who had undergone percutaneous coronary intervention for AMI were randomly assigned (1:1) to receive intravenous Stempeucel or placebo and were followed for 2 years. Results. The number of treatment-emergent adverse events observed were 18 and 21 in the Stempeucel and placebo groups, respectively. None of the adverse events were related to Stempeucel according to the investigators and independent data safety monitoring board. There was no serious adverse event in the Stempeucel group and there were three serious adverse events in the placebo group, of which one had a fatal. outcome. Ejection fraction determined by use of echocardiography showed improvement in both Stempeucel (43.06% to 47.80%) and placebo (43.44% to 45.33%) groups at 6 months (P = 0.26). Perfusion scores measured by use of single-photon emission tomography and infarct volume measured by use of magnetic resonance imaging showed no significant differences between the two groups at 6 months. Conclusions. This study showed that Stempeucel was safe and well tolerated when administered intravenously in AMI patients 2 days after percutaneous coronary intervention. The optimal dose and route of administration needs further evaluation in larger clinical trials (http://clinicaltrials.gov/show/NCT00883727).