两阶段估计法在肿瘤临床试验转组分析中的应用

doi:10.12092/j.issn.1009-2501.2021.04.006

中国临床药理学与治疗学 ›› 2021, Vol. 26 ›› Issue (4): 395-400.doi: 10.12092/j.issn.1009-2501.2021.04.006

两阶段估计法在肿瘤临床试验转组分析中的应用

于全骥，倪森淼，杨旻，仲子航，周佳薇，蔡丽馨，柏建岭，于浩

南京医科大学公共卫生学院生物统计学系，南京 211166，江苏

收稿日期:2020-12-31 修回日期:2021-03-11 出版日期:2021-04-26 发布日期:2021-05-11
通讯作者: 于浩，女，教授，研究方向：临床试验中的统计理论与方法。 E-mail: haoyu@njmu.edu.cn 柏建岭，男，副教授，研究方向：临床试验中的统计理论与方法。 E-mail: jbai@njmu.edu.cn
作者简介:于全骥，男，硕士研究生，研究方向：临床试验统计设计研究。 E-mail: yuqj@njmu.edu.cn 倪森淼，男，硕士研究生，并列第一作者，研究方向：临床试验中的统计理论与方法。 E-mail: senmiaoni@njmu.edu.cn
基金资助:
国家自然科学基金（81773554）；国家自然科学基金青年基金（81302512）

Two-stage estimation on adjustment for cross-over in oncology trials

YU Quanji, NI Senmiao, YANG Min, ZHONG Zihang, ZHOU Jiawei, CAI Lixin, BAI Jianling, YU Hao

Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, Jiangsu，China

Received:2020-12-31 Revised:2021-03-11 Online:2021-04-26 Published:2021-05-11

摘要/Abstract

摘要： 目的:探讨两阶段估计法（two-stage estimation, TSE）在肿瘤临床试验转组分析中的应用。方法:阐述TSE法的理论与实现步骤，并应用该法对一项双臂抗肿瘤药物随机对照试验的数据进行校正，评估校正前后两组生存曲线以及风险比的变化情况。将TSE法的分析结果与秩保持结构失效时间模型（rank preserving structural failure time models, RPSFT）的分析结果进行对比。结果:TSE方法校正后的分析结果显示，安慰剂组校正前的中位生存时间高于校正后的中位生存时间，同时校正前组间风险比也高于校正后的组间风险比，且与秩保持结构失效时间模型的校正结果相近。结论:TSE法易于实现且较为可靠，在临床试验转组分析中有较大的应用价值，但同时亦需注意其应用条件。

关键词: 转组, 两阶段估计法, 加速失效时间模型

Abstract: AIM: To investigate the application of two-stage estimation (TSE) on adjustment for treatment switch in oncology trials. METHODS: The theory and implementation of TSE method was described, and was applied to adjust the data from a two-arm randomized controlled trial of anti-tumor drugs. The changes of survival curves and hazard ratio of two groups after adjustment for cross-over were evaluated. In addition, the results of two-stage estimation and rank preserving structural failure time model (RPSFT) were compared. RESULTS: After adjustment for cross-over using TSE methods, the results showed that the median survival time of control group was shorter than the original one, and the hazard ratio was lower than the observed value. Moreover, TSE method showed similar results to rank preserving structural failure time model. CONCLUSION: The TSE method is relatively simple to use, reliable and has a good practice property in cross-over analysis of oncology trials. At the same time, it is necessary to pay attention to its application scopes.

Key words: cross-over, two-stage estimation, accelerated failure time model

中图分类号:

R969.1

于全骥, 倪森淼, 杨旻, 仲子航, 周佳薇, 蔡丽馨, 柏建岭, 于浩. 两阶段估计法在肿瘤临床试验转组分析中的应用[J]. 中国临床药理学与治疗学, 2021, 26(4): 395-400.

YU Quanji, NI Senmiao, YANG Min, ZHONG Zihang, ZHOU Jiawei, CAI Lixin, BAI Jianling, YU Hao. Two-stage estimation on adjustment for cross-over in oncology trials[J]. Chinese Journal of Clinical Pharmacology and Therapeutics, 2021, 26(4): 395-400.

两阶段估计法在肿瘤临床试验转组分析中的应用

Two-stage estimation on adjustment for cross-over in oncology trials

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