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作者:

Wang, Weizhen (Wang, Weizhen.) | Yin, Huan (Yin, Huan.) | Zhang, Zhongzhan (Zhang, Zhongzhan.) (学者:张忠占)

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摘要:

To assess the effectiveness of a treatment in phase II clinical trial for cancer study, an adaptive multi-stage design, especially a two-stage one, is commonly used. This type of design allows an on-going study to end early for the participant's safety and design's efficiency. Since a large value of the response rate p for the treatment is wanted, lower one-sided confidence intervals for p are of interest. Due to the limited sample size, exact intervals with a guaranteed confidence level are derived using a rank function that is based on the Clopper-Pearson lower confidence limit. When the sample size in stage 2 is a constant, two kinds of smallest intervals are constructed with or without using the sufficiency principle. The proposed intervals outperform the existing exact intervals, and the intervals not based on minimal sufficient statistic are recommended for practice due to their small expected lengths. When the sample size in stage 2 varies, the smallest interval is also proposed.

关键词:

Clopper-Pearson interval coverage probability lower one-sided confidence limit phase II clinical trial sufficiency principle

作者机构:

  • [ 1 ] [Wang, Weizhen]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 2 ] [Yin, Huan]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 3 ] [Zhang, Zhongzhan]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 4 ] [Wang, Weizhen]Wright State Univ, Dept Math & Stat, Dayton, OH 45435 USA

通讯作者信息:

  • [Wang, Weizhen]Wright State Univ, Dept Math & Stat, Dayton, OH 45435 USA

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来源 :

STATISTICAL METHODS IN MEDICAL RESEARCH

ISSN: 0962-2802

年份: 2019

期: 6

卷: 29

页码: 1682-1699

2 . 3 0 0

JCR@2022

ESI学科: MATHEMATICS;

ESI高被引阀值:25

JCR分区:3

被引次数:

WoS核心集被引频次: 4

SCOPUS被引频次: 3

ESI高被引论文在榜: 0 展开所有

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