Sample size determination in group-sequential clinical trials with two co-primary endpoints

被引：17

作者：

Asakura, Koko ^{[1
,2
]}

Hamasaki, Toshimitsu ^{[1
,2
]}

Sugimoto, Tomoyuki ^{[3
]}

Hayashi, Kenichi ^{[1
]}

Evans, Scott R. ^{[4
,5
]}

Sozu, Takashi ^{[6
]}

机构：

[1] Osaka Univ, Grad Sch Med, Dept Biomed Stat, Osaka, Japan

[2] Natl Cerebral & Cardiovasc Ctr, Res & Dev Initiat Ctr, Off Biostat & Data Management, Suita, Osaka 5658565, Japan

[3] Hirosaki Univ, Grad Sch Sci & Technol, Dept Math Sci, Aomori, Japan

[4] Harvard Univ, Sch Publ Hlth, Dept Biostat, Boston, MA 02115 USA

[5] Harvard Univ, Sch Publ Hlth, Ctr Biostat AIDS Res, Boston, MA 02115 USA

[6] Kyoto Univ, Sch Publ Hlth, Dept Biostat, Kyoto, Japan

来源：

STATISTICS IN MEDICINE | 2014年 / 33卷 / 17期

基金：

美国国家卫生研究院;

关键词：

average sample number; conditional power; Cui-Hung-Wang statistics; co-primary endpoints; group-sequential methods; maximum sample size; sample size recalculation; Type I error; ADAPTIVE EXTENSIONS; TESTING PRIMARY; EFFICACY; DISEASE; DESIGN; POWER;

D O I：

10.1002/sim.6154

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

We discuss sample size determination in group-sequential designs with two endpoints as co-primary. We derive the power and sample size within two decision-making frameworks. One is to claim the test intervention's benefit relative to control when superiority is achieved for the two endpoints at the same interim timepoint of the trial. The other is when superiority is achieved for the two endpoints at any interim timepoint, not necessarily simultaneously. We evaluate the behaviors of sample size and power with varying design elements and provide a real example to illustrate the proposed sample size methods. In addition, we discuss sample size recalculation based on observed data and evaluate the impact on the power and Type I error rate. Copyright (C) 2014 John Wiley & Sons, Ltd.

引用

页码：2897 / 2913

页数：17

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