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N Engl J Med. 2018 Dec 13;379(24):2297-2306. doi: 10.1056/NEJMoa1805374. Epub 2018 Aug 27.
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Cell therapy trials for heart regeneration - lessons learned and future directions.心脏再生的细胞治疗临床试验——经验教训和未来方向。
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较大的舒张末期容积与非缺血性扩张型心肌病患者对细胞治疗的反应相关。

Larger End-Diastolic Volume Associates With Response to Cell Therapy in Patients With Nonischemic Dilated Cardiomyopathy.

机构信息

Advanced Heart Failure and Transplantation Center, UMC Ljubljana, Slovenia.

Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA.

出版信息

Mayo Clin Proc. 2020 Oct;95(10):2125-2133. doi: 10.1016/j.mayocp.2020.02.031.

DOI:10.1016/j.mayocp.2020.02.031
PMID:33012343
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7539130/
Abstract

OBJECTIVE

To investigate the association of left ventricular end-diastolic volume (LVEDV) and the response to cell therapy in patients with nonischemic dilated cardiomyopathy (NICM).

PATIENTS AND METHODS

Five-year registry data from 133 consecutive patients with NICM who underwent CD34 cell treatment were analyzed. All patients received granulocyte-colony stimulating factor; CD34 cells were collected by apheresis and delivered by transendocardial injections. Patients with baseline LVEDV less than 200 mL (group A; n=72) and patients with LVEDV 200 to 370 mL (group B; n=54) were included. Patients with LVEDV greater than 370 mL were excluded (n=7). Favorable ejection fraction response was pre-defined by improvement in left ventricular ejection fraction (LVEF) greater than or equal to 5% at 1 y post-cell therapy.

RESULTS

At baseline, groups A and B were comparable with regards to age (52±11 y in group A vs 53±10 y in group B; P=.95), sex (male: 79% vs 83%, respectively; P=.55), creatinine (1.07±0.28 mg/dL vs 1.03±0.21 mg/dL, respectively; P=.21), or N-terminal probrain natriuretic peptide (1454±1658 pg/mL vs 1589±1338 pg/mL, respectively; P=.80). Baseline LVEF was higher in group A (32.8±8.7%) than in group B (30.2±8.7%; P=.03). During follow-up, there were four deaths in group A (5.6%), and 2 in group B (3.7%, P=.63). At 1-year post-cell therapy, LVEDV decreased significantly in group B (-56±30 mL; P=.003), but not in group A (+12±97 mL; P=.13). On multivariate analysis, baseline LVEDV was an independent correlate of favorable response in LVEF to therapy (P=.02).

CONCLUSION

Larger LVEDV was associated with more pronounced increase in LVEF after transendocardial CD34 cell therapy in NICM patients, informing target individuals with the highest likelihood of regenerative response.

TRIAL REGISTRATION

clinicaltrials.gov identifier: NCT02445534.

摘要

目的

探讨左心室舒张末期容积(LVEDV)与非缺血性扩张型心肌病(NICM)患者细胞治疗反应的关系。

方法

对 133 例连续接受 CD34 细胞治疗的 NICM 患者的 5 年登记数据进行分析。所有患者均接受粒细胞集落刺激因子治疗;CD34 细胞通过体外分离术采集,并通过心内膜内注射给药。将基线 LVEDV 小于 200mL 的患者纳入 A 组(n=72),LVEDV 为 200-370mL 的患者纳入 B 组(n=54),LVEDV 大于 370mL 的患者排除在外(n=7)。将左心室射血分数(LVEF)增加大于等于 5%定义为细胞治疗后 1 年时射血分数的有利反应。

结果

基线时,A 组和 B 组在年龄(A 组 52±11 岁,B 组 53±10 岁;P=.95)、性别(男性:分别为 79%和 83%;P=.55)、肌酐(1.07±0.28mg/dL 与 1.03±0.21mg/dL;P=.21)或 N 末端脑利钠肽前体(1454±1658pg/mL 与 1589±1338pg/mL;P=.80)方面无差异。A 组的基线 LVEF 高于 B 组(32.8±8.7%与 30.2±8.7%;P=.03)。随访期间,A 组有 4 例死亡(5.6%),B 组有 2 例(3.7%;P=.63)。细胞治疗后 1 年,B 组的 LVEDV 明显降低(-56±30mL;P=.003),但 A 组无明显变化(+12±97mL;P=.13)。多变量分析显示,基线 LVEDV 是治疗后 LVEF 反应良好的独立相关因素(P=.02)。

结论

在 NICM 患者中,心内膜内 CD34 细胞治疗后,较大的 LVEDV 与 LVEF 更明显的增加相关,提示具有最高再生反应可能性的目标个体。

试验注册

clinicaltrials.gov 标识符:NCT02445534。