当代癌症 III 期随机对照试验中具有时间事件结局的统计检验/估计方法。
Statistical Test/Estimation Methods Used in Contemporary Phase III Cancer Randomized Controlled Trials with Time-to-Event Outcomes.
机构信息
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
Department of Data Sciences, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
出版信息
Oncologist. 2020 Feb;25(2):91-93. doi: 10.1634/theoncologist.2019-0464. Epub 2019 Nov 19.
Abstract
There exist a wide variety of statistical methods for clinical trials. Despite decades of clinical trial research, little is known about which methods are used in data analysis practice of the real world. This review describes the evidence of existing practices on survival data analysis and identifies potential opportunities to improve them.
摘要
临床试验中存在着各种各样的统计方法。尽管已经进行了几十年的临床试验研究，但对于在现实世界的数据分析实践中使用哪些方法，人们知之甚少。本综述描述了生存数据分析中现有实践的证据，并确定了改进这些实践的潜在机会。
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2
Treatment effects measured by restricted mean survival time in trials of immune checkpoint inhibitors for cancer.免疫检查点抑制剂治疗癌症的临床试验中通过受限平均生存时间衡量的治疗效果。
Ann Oncol. 2018 May 1;29(5):1320-1324. doi: 10.1093/annonc/mdy075.
3
Cancer Biology and Survival Analysis in Cancer Trials: Restricted Mean Survival Time Analysis versus Hazard Ratios.癌症生物学与癌症试验中的生存分析：限制平均生存时间分析与风险比。
Clin Oncol (R Coll Radiol). 2018 Sep;30(9):e75-e80. doi: 10.1016/j.clon.2018.04.011.
4
Comparison of the restricted mean survival time with the hazard ratio in superiority trials with a time-to-event end point.在具有事件发生时间终点的优效性试验中，受限平均生存时间与风险比的比较。
Pharm Stat. 2018 May;17(3):202-213. doi: 10.1002/pst.1846. Epub 2017 Dec 28.
5
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6
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