单臂临床试验生存分析的样本量估计

目的 提出并验证单臂临床试验进行生存分析时的样本量估计方法。方法 通过理论公式的推导,得到单臂临床试验进行生存分析时样本量估计的计算公式,并采用STATA软件进行蒙特卡洛随机模拟,估计实际的检验效能,从而验证公式的准确性。结果 随机模拟结果表明在α=0.025和α=0.01的情况下,用本文提出的公式计算得到的样本量模拟得到的检验效能与预设的检验效能基本一致。结论 在单臂临床试验中利用本文的公式计算得到的样本含量能够达到预定的检验效能。本研究的成果为抗肿瘤药物的早期临床研究的样本量估计提供了可靠的依据。...

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Bibliographic Details
Published in复旦学报（医学版） Vol. 44; no. 4; pp. 517 - 520
Main Author 张秀萍 赵耐青
Format Journal Article
LanguageChinese
Published 复旦大学公共卫生学院生物统计学教研室 上海 200032 2017
Subjects
单臂临床试验
样本量估计
生存分析
single-arm trials
单臂临床试验
survival analysis
样本量估计
sample size estimation
生存分析
Online AccessGet full text
ISSN1672-8467
DOI10.3969/j.issn.1672-8467.2017.04.022

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Summary:目的 提出并验证单臂临床试验进行生存分析时的样本量估计方法。方法 通过理论公式的推导,得到单臂临床试验进行生存分析时样本量估计的计算公式,并采用STATA软件进行蒙特卡洛随机模拟,估计实际的检验效能,从而验证公式的准确性。结果 随机模拟结果表明在α=0.025和α=0.01的情况下,用本文提出的公式计算得到的样本量模拟得到的检验效能与预设的检验效能基本一致。结论 在单臂临床试验中利用本文的公式计算得到的样本含量能够达到预定的检验效能。本研究的成果为抗肿瘤药物的早期临床研究的样本量估计提供了可靠的依据。
Bibliography:single-arm trials; sample size estimation; survival analysis
ZHANG Xiu-ping, ZHAO Nai-qing （ Department of Biostatistics, School of Public Health, Fudan University, Shanghai 200032, China）
Objective To propose a method to calculate sample size for survival analysis in single-arm trials. Methods Through theoretical formula derivation, we provided sample size estimation method for survival analysis in single-arm trials. Monte-Carlo simulations using STATA software were conducted to obtain the empirical power, so as to verify the accuracy of the estimated sample size. Results Simulation results showed that, when α = 0. 025 and α = 0.01, the power of the sample size calculated by the formula is in accordance with the predetermined power. Conclusions The formula of sample size estimation we proposed could achieve the predetermined power for survival analysis in single-arm trials. Findings of this study provide reliable basis for sample size estimation of early stage antineoplastic drug clinical studies.
31-1885/R
ISSN:1672-8467
DOI:10.3969/j.issn.1672-8467.2017.04.022