Challenges in the harmonization of immune monitoring studies and trial design for cell-based therapies in the context of hematopoietic cell transplantation for pediatric cancer patients.
Challenges in the harmonization of immune monitoring studies and trial design for cell-based therapies in the context of hematopoietic cell transplantation for pediatric cancer patients.
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DOI：
10.1016/j.jcyt.2015.09.008
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发表时间：
2015-12
期刊：
影响因子：
4.5
通讯作者：
Westhafen Intercontinental Group
中科院分区：
文献类型：
--
作者：
Nierkens S;Lankester AC;Egeler RM;Bader P;Locatelli F;Pulsipher MA;Bollard CM;Boelens JJ;Westhafen Intercontinental Group
旨在通过辅助性细胞干预改善儿童造血细胞移植(HCT)结果的临床试验受到数量少和儿科特有特征的限制。由于需要更多的儿科HCT中心参与试验，因此需要协调针对疾病的临床试验和免疫监测。到目前为止，大多数I/II期试验选择在不同时间点进行评估的不同终点，这使得研究间比较很困难，有时甚至是不可能的。在这篇综述中，我们讨论了协调临床试验设计需要考虑的各个方面，以及在HCT背景下基于细胞的干预试验中标准化(免疫)监测方案的关键要素。来自应用协调试验设计的试验的比较数据将导致优化的免疫治疗方案，以最大限度地提高临床疗效，同时将毒性降至最低。
Clinical trials aimed at improving results of hematopoietic cell transplantation (HCT) by adjuvant cell-based interventions in children have been limited by small numbers and pediatric-specific features. The need for a larger number of pediatric HCT centers to participate in trials has resulted in a demand for harmonization of disease-specific clinical trials and immune-monitoring. Thus far, most phase I/II trials select different end points evaluated at disparate time points, making inter-study comparisons difficult and, sometimes, impossible. In this review, we discuss the various aspects that are important to consider for harmonizing clinical trial design as well as the critical elements for standardized (immune)-monitoring protocols in cell-based intervention trials in the context of HCT. Comparison data from trials applying harmonized trial design will lead to optimized immunotherapeutic treatment protocols to maximize clinical efficacy while minimizing toxicity.
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影响因子：
20.3
作者：
Pidala, Joseph;Lee, Stephanie J.;Anasetti, Claudio
通讯作者：
Anasetti, Claudio
影响因子：
20.3
作者：
Pulsipher, Michael A.;Carlson, Chris;Grupp, Stephan A.
通讯作者：
Grupp, Stephan A.
DOI：
10.1126/science.1198704
发表时间：
2011-05-06
期刊：
Science (New York, N.Y.)
影响因子：
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作者：
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影响因子：
20.3
作者：
Leen, Ann M.;Bollard, Catherine M.;Heslop, Helen E.
通讯作者：
Heslop, Helen E.
影响因子：
11.5
作者：
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通讯作者：
Whiteside, Theresa L.