用于二元结局随机试验的两阶段设计。

A two-stage design for randomized trials with binary outcomes.

作者信息

Wieand S, Therneau T

出版信息

Control Clin Trials. 1987 Mar;8(1):20-8. doi: 10.1016/0197-2456(87)90022-5.

Abstract

A two-stage rule for a randomized clinical trial with a dichotomous outcome is presented. It is based on a rule discussed by Ellenberg and Eisenberger (Cancer Treat Rep 69:1147-1154, 1985). The rule is shown to lead to a reduction in expected sample size if the test treatment is ineffective, with almost no loss in power. All the mathematical formulas required to design such a rule are given, as well as some time-saving approximations. The accuracy of the approximations is shown.

摘要

本文提出了一种用于二分类结局随机临床试验的两阶段规则。它基于Ellenberg和Eisenberger（《癌症治疗报告》69:1147 - 1154, 1985）所讨论的规则。结果表明，如果试验治疗无效，该规则可减少预期样本量，且功效几乎没有损失。给出了设计此类规则所需的所有数学公式以及一些节省时间的近似方法。展示了这些近似方法的准确性。