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中国临床药理学与治疗学 ›› 2017, Vol. 22 ›› Issue (8): 917-921.

• 定量药理学 • 上一篇    下一篇

单组目标值临床试验多指标时的样本量估计

陆梦洁1,刘玉秀2,卢光明1,张龙江1,黄 伟1,葛爱晨3   

  1. 1中国人民解放军南京总医院(金陵医院)医学影像中心, 2中国人民解放军南京总医院(金陵医院)医学统计学教研室, 3南京医科大学医政学院,南京 210002,江苏
  • 收稿日期:2017-03-07 修回日期:2017-04-06 出版日期:2017-08-26 发布日期:2017-08-18
  • 通讯作者: 刘玉秀,男,硕士,主任医师,教授,硕士生导师,研究方向:临床试验生物统计学方法学研究及应用。 E-mail: liu_yuxiu@163.com
  • 作者简介:陆梦洁,女,硕士,研究方向:临床试验生物统计学方法学研究。 E-mail: 898545969@qq.com
  • 基金资助:

    国家自然科学基金项目(81473066)

Sample size calculation for single-arm OPC trials with multi endpoints

LU Mengjie 1, LIU Yuxiu 2, LU Guangming 1, ZHANG Longjiang 1, HUANG Wei 1, GE Aichen 3   

  1. 1 Medical Imaging Center, Jinling Hospital, 2 Department of Medical Statistics, JinlingHospital, 3 School of Health Policy & Management, Nanjing Medical University, Nanjing 210002, Jiangsu, China
  • Received:2017-03-07 Revised:2017-04-06 Online:2017-08-26 Published:2017-08-18

摘要:

目的: 针对多个以率为共同终点评价指标的单组临床试验,探讨控制全局把握度的样本量估计方法。方法: 根据单组目标值法单指标时的样本量估计方法,在理论上阐明单组临床试验多个共同指标时样本量估计的原理,提出控制全局把握度的样本量估计方法。事先设定各单指标目标值、靶值、I类错误率及总的II类错误率水平,在给定的样本量下可算得各单指标对应的把握度,相乘后即为全局把握度,利用逐步寻值即可获得达到全局把握度时所需的样本量。结果: 结合一个单组临床试验案例,基于4个率指标,利用样本量估计的正态近似法和确切概率法,分析对比了传统直接校正把握度的方法和本文逐步寻值法所估计的样本量结果,后者能更准确地控制全局把握度,所需的样本量更少。结论: 本文给出的单组目标值临床试验多指标情形下逐步寻值的样本量估计方法,能较好地控制全局把握度、节约样本量,具有很强的实用价值。

关键词: 目标值法, 多指标, 样本量, 全局把握度, 逐步寻值法

Abstract:

AIM: To explore the sample size calculation by control global power for multiple rates as multi endpoints in single-arm Objective Performance Criteria (OPC) trials. METHODS: Based on the sample size calculations for single-arm OPC with single endpoint, we illustrated the statistical principle of sample size calculations with multi endpoints in theory and proposed a method by control global power. According to the pre-determined level of OPC, target value, type I error and total type II error, the power of each endpoint could be evaluated under the given sample sizes and the multiplication of all powers was the actual global power. The sample size reached objective global power was evaluated by the stepwise search method. RESULTS: We compared the sample sizes using the conventional direct power correction and stepwise search method through an example of one-sample clinical trial with four proportions based on normal approximation method and exact method. The results showed that the latter could more accurately control the global power and reduced the sample sizes. CONCLUSION: We proposed a solution to correct sample sizes for single-arm OPC trials with multi endpoints, which could control global power and reduce sample sizes significantly.

Key words: objective performance criteria, multi endpoints, sample size, global power, stepwise search method

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