血液肿瘤学试验中的Estimats

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摘要翻译：
ICH E9指南增编中的估计和框架有助于讨论，以确保感兴趣的关键问题、分析和解释之间的一致性。治疗知识和药物机制在决定策略、定义估计和临床试验设计中起着至关重要的作用。针对血液系统恶性肿瘤患者的临床试验常常给试验设计带来独特的挑战，这是由于治疗方案的复杂性和存在潜在的治愈但高风险的程序，如干细胞移植或跨不同阶段（诱导、巩固、维持）的治疗顺序。在这里，我们说明如何在血液学临床试验中应用评估和框架，以及评估和框架如何解决试验结果解释的潜在困难。这篇论文是一个跨行业合作的结果，将国际协调会议(ICH)E9附录的概念与应用程序联系起来。三个随机3期试验将被用来考虑共同的挑战，包括血液肿瘤试验中的并发事件，以说明不同的科学问题和试验设计、数据收集、分析和解释的估计和选择的后果。在研究方案和统计分析计划中描述估计和的模板语言建议供统计学家参考。
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英文标题：
《Estimands in Hematologic Oncology Trials》
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作者：
Steven Sun and Hans-Jochen Weber and Emily Butler and Kaspar Rufibach
  and Satrajit Roychoudhury
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最新提交年份：
2020
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分类信息：

一级分类：Quantitative Biology        数量生物学
二级分类：Other Quantitative Biology        其他定量生物学
分类描述：Work in quantitative biology that does not fit into the other q-bio classifications
不适合其他q-bio分类的定量生物学工作
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一级分类：Statistics        统计学
二级分类：Methodology        方法论
分类描述：Design, Surveys, Model Selection, Multiple Testing, Multivariate Methods, Signal and Image Processing, Time Series, Smoothing, Spatial Statistics, Survival Analysis, Nonparametric and Semiparametric Methods
设计，调查，模型选择，多重检验，多元方法，信号和图像处理，时间序列，平滑，空间统计，生存分析，非参数和半参数方法
--

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英文摘要：
  The estimand framework included in the addendum to the ICH E9 guideline facilitates discussions to ensure alignment between the key question of interest, the analysis, and interpretation. Therapeutic knowledge and drug mechanism play a crucial role in determining the strategy and defining the estimand for clinical trial designs. Clinical trials in patients with hematological malignancies often present unique challenges for trial design due to complexity of treatment options and existence of potential curative but highly risky procedures, e.g. stem cell transplant or treatment sequence across different phases (induction, consolidation, maintenance). Here, we illustrate how to apply the estimand framework in hematological clinical trials and how the estimand framework can address potential difficulties in trial result interpretation.   This paper is a result of a cross-industry collaboration to connect the International Conference on Harmonisation (ICH) E9 addendum concepts to applications. Three randomized phase 3 trials will be used to consider common challenges including intercurrent events in hematologic oncology trials to illustrate different scientific questions and the consequences of the estimand choice for trial design, data collection, analysis, and interpretation. Template language for describing estimand in both study protocols and statistical analysis plans is suggested for statisticians' reference.
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PDF链接：
https://arxiv.org/pdf/2010.00957

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关键词：Tima Mat ima Est Tim 评估 临床试验 challenges potential 框架