Alignment of practices for data harmonization across multi-center cell therapy trials: a report from the Consortium for Pediatric Cellular Immunotherapy.
Alignment of practices for data harmonization across multi-center cell therapy trials: a report from the Consortium for Pediatric Cellular Immunotherapy.
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DOI：
10.1016/j.jcyt.2021.08.007
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发表时间：
2022-03
期刊：
影响因子：
4.5
通讯作者：
Wilson AL
中科院分区：
文献类型：
--
作者：
Abdel-Azim H;Dave H;Jordan K;Rawlings-Rhea S;Luong A;Wilson AL
免疫效应细胞(IEC)疗法彻底改变了我们治疗复发性B细胞恶性肿瘤的方法，对IEC研究用途的兴趣正在迅速扩展到其他疾病。目前IEC疗法分析面临的挑战包括样本量小，临床试验机会有限，以及缺乏与IEC疗法相关的疗效和毒性预测生物标记物。回溯性和前瞻性多中心细胞治疗试验可以通过协调临床终点和免疫监测的相关分析来帮助克服这些障碍，允许额外的交叉试验分析来确定失败和成功的生物标志物。儿科细胞免疫治疗联盟(CPCI)提供了一个独特的平台，通过多中心临床试验为儿童提供尖端的细胞和基因治疗，以应对上述挑战。在这里，我们讨论一些重要的分析前变量，如生物样品采集和初始处理程序，这些变量影响在参与CPCI站点的IEC试验中常用的生物标记物分析。我们回顾了最新的文献，并提供了数据来支持实践的调整和标准化建议，这些实践可能会影响流式细胞术检测免疫效应器功能，并可能影响细胞因子/趋化因子数据的解释。我们还确定了经常使试验之间的平行比较变得困难或不可能的关键差距。
Immune effector cell (IEC) therapies have revolutionized our approach to relapsed B cell malignancies, and interest in the investigational use of IEC is rapidly expanding into other diseases. Current challenges in the analysis of IEC therapies include small sample sizes, limited access to clinical trials, and a paucity of predictive biomarkers of efficacy and toxicity associated with IEC therapies. Retrospective and prospective multi-center cell therapy trials can assist in overcoming these barriers through harmonization of clinical endpoints and correlative assays for immune monitoring, allowing additional cross trial analysis to identify biomarkers of failure and success. The Consortium for Pediatric Cellular Immunotherapy (CPCI) offers a unique platform to address the challenges described above by delivering cutting-edge cell and gene therapies for children through multi-center clinical trials. Here we discuss some of the important pre-analytic variables, such as biospecimen collection and initial processing procedures, that affect biomarker assays commonly used in IEC trials across participating CPCI sites. We review the recent literature and provide data to support recommendations for alignment and standardization of practices that can affect flow cytometry assays measuring immune effector function and that can affect interpretation of cytokine/chemokine data. We also identify critical gaps that often make parallel comparisons between trials difficult or impossible.
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影响因子：
20.3
作者：
Gardner, Rebecca A.;Finney, Olivia;Jensen, Michael C.
通讯作者：
Jensen, Michael C.
多中心细胞疗法临床试验的加工实验室注意事项：小儿细胞免疫疗法财团的报告。
影响因子：
4.5
作者：
Lindgren C;Leinbach A;Annis J;Tanna J;Zhang N;Esensten JH;Hanley PJ
通讯作者：
Hanley PJ
影响因子：
2.9
作者：
Fenizia F;Pasquale R;Abate RE;Lambiase M;Roma C;Bergantino F;Chaudhury R;Hyland F;Allen C;Normanno N
通讯作者：
Normanno N
影响因子：
4.5
作者：
Nierkens S;Lankester AC;Egeler RM;Bader P;Locatelli F;Pulsipher MA;Bollard CM;Boelens JJ;Westhafen Intercontinental Group
通讯作者：
Westhafen Intercontinental Group
影响因子：
5.6
作者：
Bent R;Moll L;Grabbe S;Bros M
通讯作者：
Bros M