论文标题
从估计和框架的角度来看，肿瘤学临床试验的响应时间和时间
Duration of and time to response in oncology clinical trials from the perspective of the estimand framework
论文作者
论文摘要
在肿瘤学早期临床研究中，响应持续时间（DOR）和响应时间（TTR）通常被评估为在肿瘤学早期临床研究中的次要终点，当疗效通过最佳总体反应（BOR）评估并表示为总体反应率（ORR）。尽管通常在单臂研究中使用了DOR和TTR，但这些终点的定义及其旨在回答的问题仍不清楚。在估算和框架的启发下，我们对DOR和TTR提出了相关的科学问题，并提出了相应的估计定义。我们详细介绍了如何处理相关的事件，这些事件应遵循与主要响应估计的相同考虑因素。地幔细胞淋巴瘤中的案例研究说明了DOR和TTR相关估计的实施。我们将论文结束，并在临床研究方案中实施DOR和TTR的实用建议。
Duration of response (DOR) and time to response (TTR) are typically evaluated as secondary endpoints in early-stage clinical studies in oncology when efficacy is assessed by the best overall response (BOR) and presented as the overall response rate (ORR). Despite common use of DOR and TTR in particular in single-arm studies, the definition of these endpoints and the questions they are intended to answer remain unclear. Motivated by the estimand framework, we present relevant scientific questions of interest for DOR and TTR and propose corresponding estimand definitions. We elaborate on how to deal with relevant intercurrent events which should follow the same considerations as implemented for the primary response estimand. A case study in mantle cell lymphoma illustrates the implementation of relevant estimands of DOR and TTR. We close the paper with practical recommendations to implement DOR and TTR in clinical study protocols.
