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自体骨骼肌母细胞片移植治疗终末期缺血性心肌病的长期疗效。

Long-term outcomes of autologous skeletal myoblast cell-sheet transplantation for end-stage ischemic cardiomyopathy.

机构信息

Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, Suita, Osaka 565-0871, Japan.

Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, Suita, Osaka 565-0871, Japan.

出版信息

Mol Ther. 2021 Apr 7;29(4):1425-1438. doi: 10.1016/j.ymthe.2021.01.004. Epub 2021 Jan 9.

DOI:10.1016/j.ymthe.2021.01.004
PMID:33429079
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8058489/
Abstract

We evaluated the cardiac function recovery following skeletal myoblast cell-sheet transplantation and the long-term outcomes after applying this treatment in 23 patients with ischemic cardiomyopathy. We defined patients as "responders" when their left ventricular ejection fraction remained unchanged or improved at 6 months after treatment. At 6 months, 16 (69.6%) patients were defined as responders, and the average increase in left ventricular ejection fraction was 4.9%. The responders achieved greater improvement degrees in left ventricular and hemodynamic function parameters, and they presented improved exercise capacity. During the follow-up period (56 ± 28 months), there were four deaths and the overall 5-year survival rate was 95%. Although the responders showed higher freedom from mortality and/or heart failure admission (5-year, 81% versus 0%; p = 0.0002), both groups presented an excellent 5-year survival rate (5-year, 93% versus 100%; p = 0.297) that was higher than that predicted using the Seattle Heart Failure Model. The stepwise logistic regression analysis showed that the preoperative estimated glomerular filtration rate and the left ventricular end-systolic volume index were independently associated with the recovery progress. Approximately 70% of patients with "no-option" ischemic cardiomyopathy responded well to the cell-sheet transplantation. Preoperative renal and left ventricular function might predict the patients' response to this treatment.

摘要

我们评估了骨骼肌母细胞片移植后心脏功能的恢复情况,并在 23 例缺血性心肌病患者中应用该治疗的长期结果。我们将左心室射血分数在治疗后 6 个月保持不变或改善的患者定义为“应答者”。在 6 个月时,16 例(69.6%)患者被定义为应答者,左心室射血分数的平均增加为 4.9%。应答者的左心室和血液动力学功能参数的改善程度更大,他们的运动能力也得到了提高。在随访期间(56±28 个月),有 4 例死亡,总 5 年生存率为 95%。尽管应答者的死亡率和/或心力衰竭入院率更高(5 年,81%对 0%;p=0.0002),但两组的 5 年生存率(5 年,93%对 100%;p=0.297)均高于西雅图心力衰竭模型预测值。逐步逻辑回归分析显示,术前估算肾小球滤过率和左心室收缩末期容积指数与恢复进程独立相关。大约 70%的“无选择”缺血性心肌病患者对细胞片移植反应良好。术前肾功能和左心室功能可能预测患者对该治疗的反应。

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