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Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage
The Stata Journal: Promoting communications on statistics and Stata ( IF 3.2 ) Pub Date : 2019-12-18 , DOI: 10.1177/1536867x19893616
Alexandra Blenkinsop 1 , Babak Choodari-Oskooei 1
Affiliation  

Royston et al.’s (2011, Trials 12: 81) multiarm, multistage (MAMS) framework for the design of randomized clinical trials uses intermediate outcomes to drop research arms early for lack of benefit at interim stages, increasing efficiency in multiarm designs. However, additionally permitting interim evaluation of efficacy on the primary outcome measure could increase adoption of the design and result in practical benefits, such as savings in patient numbers and cost, should any efficacious arm be identified early. The nstage command, which aids the design of MAMS trial designs, has been updated to support this methodological extension. Operating characteristics can now be calculated for a design with binding or nonbinding stopping rules for lack of benefit and with efficacy stopping boundaries. An additional option searches for a design that strongly controls the familywise error rate at the desired level. We illustrate how the new features can be used to design a trial with the drop-down menu, using the original comparisons from the MAMS trial STAMPEDE as an example. The new functionality of the command will serve a broader range of trial objectives and increase efficiency of the design and thus increase uptake of the MAMS design in practice.



中文翻译:

多臂,多阶段随机对照试验,因疗效和缺乏获益而有界限:对nstage的更新

Royston等人(2011,Trials 12:81)的多臂,多阶段(MAMS)框架用于随机临床试验的设计,由于中间阶段缺乏获益,因此采用中间结果来尽早放弃研究小组,从而提高了多臂设计的效率。但是,如果可以及早发现任何有效的手臂,则另外允许对主要结局指标进行疗效的中期评估可能会增加设计的采用率,并带来实际好处,例如节省患者人数和成本。该nstage辅助MAMS试验设计设计的命令已更新,以支持此方法扩展。现在可以针对具有约束或非约束停止规则的设计(缺乏效益)和功效停止边界来计算操作特性。另一个选项可搜索可将家族错误率严格控制在所需水平的设计。我们以MAMS试用STAMPEDE中的原始比较为例,说明如何使用新功能通过下拉菜单设计试验。该命令的新功能将服务于更广泛的试验目标,并提高设计效率,从而在实践中增加对MAMS设计的采用。

更新日期:2019-12-18
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