Defining the optimal historical control group for a phase 1 trial of mesenchymal stromal cell delivery through cardiopulmonary bypass in neonates and infants.
Defining the optimal historical control group for a phase 1 trial of mesenchymal stromal cell delivery through cardiopulmonary bypass in neonates and infants.
复制标题
为新生儿和婴儿通过心肺旁路输送间充质基质细胞的一期试验定义最佳历史对照组。
DOI：
10.1017/s1047951122002633
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发表时间：
2023
影响因子：
1
通讯作者：
Ishibashi,Nobuyuki
中科院分区：
文献类型：
--
作者：
Kobayashi,Kei;Higgins,Tessa;Liu,Christopher;Ayodeji,Mobolanle;Wernovsky,Gil;Jonas,RichardA;Ishibashi,Nobuyuki
小儿心脏手术中通过心脏搭桥术进行间充质基质细胞输送研究是一项前瞻性、开放标签、单中心、剂量递增的1期试验，评估在心脏手术期间向新生儿/婴儿输送间充质基质细胞的安全性/可行性。将结果与类似人群的历史数据进行比较。我们的目的是确定一个最佳的对照组，用于在小儿心脏手术试验中通过心脏搭桥进行间充质基质细胞输送。方法2015年至2020年期间，在生命的前6个月内接受了双心室修复术而没有主动脉弓重建的连续患者使用相同的纳入/排除标准为在儿科心脏手术试验中通过心脏旁路输送间充质基质细胞的I期试验（n = 169）。患者被分为三个诊断组之一：室间隔缺损型，法洛四联症型，大动脉转位型。为了确定年龄效应，将患者分为两组进行分析：A组（2015-2017年）和B组（2018-2020年）。除了生物标志物，三个术后评分方法（变力性和血管活性变力性评分和儿科死亡风险-III）进行了assessed.ResultsAll值为三个评分系统的心脏异常的复杂性是一致的。所有诊断组之间的最大变力性和血管活性变力性评分显示出显著差异，证实了高灵敏度。尽管时代组之间的手术因素没有差异，我们观察到较低的肌力和血管活性肌力评分在组B，符合改善手术后的过程中，近年来在我们的centre.ConclusionsOur研究确认最大肌力和血管活性肌力评分作为重要的定量措施后，新生儿/婴儿心脏手术。应在诊断分组内比较临床结局。最佳对照组应该只包括最近的病人。这项初步研究将有助于确定未来疗效/有效性研究的样本量。
ObjectiveThe Mesenchymal Stromal Cell Delivery through Cardiopulmonary Bypass in Pediatric Cardiac Surgery study is a prospective, open-label, single-centre, dose-escalation phase 1 trial assessing the safety/feasibility of delivering mesenchymal stromal cells to neonates/infants during cardiac surgery. Outcomes will be compared with historical data from a similar population. We aim to define an optimal control group for use in the Mesenchymal Stromal Cell Delivery through Cardiopulmonary Bypass in Pediatric Cardiac Surgery trial.MethodsConsecutive patients who underwent a two-ventricle repair without aortic arch reconstruction within the first 6 months of life between 2015 and 2020 were studied using the same inclusion/exclusion criteria as the Phase 1 Mesenchymal Stromal Cell Delivery through Cardiopulmonary Bypass in Pediatric Cardiac Surgery trial (n = 169). Patients were allocated into one of three diagnostic groups: ventricular septal defect type, Tetralogy of Fallot type, and transposition of the great arteries type. To determine era effect, patients were analysed in two groups: Group A (2015–2017) and B (2018–2020). In addition to biological markers, three post-operative scoring methods (inotropic and vasoactive-inotropic scores and the Pediatric Risk of Mortality-III) were assessed.ResultsAll values for three scoring systems were consistent with complexity of cardiac anomalies. Max inotropic and vasoactive-inotropic scores demonstrated significant differences between all diagnosis groups, confirming high sensitivity. Despite no differences in surgical factors between era groups, we observed lower inotropic and vasoactive-inotropic scores in group B, consistent with improved post-operative course in recent years at our centre.ConclusionsOur studies confirm max inotropic and vasoactive-inotropic scores as important quantitative measures after neonatal/infant cardiac surgery. Clinical outcomes should be compared within diagnostic groupings. The optimal control group should include only patients from a recent era. This initial study will help to determine the sample size of future efficacy/effectiveness studies.