Advantages of multi-arm non-randomised sequentially allocated cohort designs for Phase II oncology trials.
Advantages of multi-arm non-randomised sequentially allocated cohort designs for Phase II oncology trials.
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DOI：
10.1038/s41416-021-01613-5
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发表时间：
2022-03
影响因子：
8.8
通讯作者：
Wason JMS
中科院分区：
文献类型：
--
作者：
Mossop H;Grayling MJ;Gallagher FA;Welsh SJ;Stewart GD;Wason JMS
需要有效的试验设计来优先考虑II期试验中有前途的药物。适应性设计是这种设计的例子，但如果在评估患者对治疗的反应时存在延迟，则其效率会降低。受肾细胞癌WIRE试验（NCT 03741426）的启发，我们比较了三种测试多个治疗组的试验方法：（1）按顺序进行的单组试验和中期分析;（2）平行多组多阶段试验和（3）WIRE中使用的设计，我们称之为多组序贯试验与有效招募（MASTER）设计。MASTER设计一次招募一个手臂的患者，当招募到中期分析所需数量时，暂停招募手臂。我们进行了一项模拟研究，以比较三种不同的试验设计需要多长时间来评估一些新的治疗组。平行多臂多级和MASTER设计比单独试验更有效。MASTER设计在存在终点延迟或募集非常快时提供额外的效率。我们推荐MASTER设计作为在非比较性II期试验中测试多种有希望的癌症治疗的有效方法。
Efficient trial designs are required to prioritise promising drugs within Phase II trials. Adaptive designs are examples of such designs, but their efficiency is reduced if there is a delay in assessing patient responses to treatment. Motivated by the WIRE trial in renal cell carcinoma (NCT03741426), we compare three trial approaches to testing multiple treatment arms: (1) single-arm trials in sequence with interim analyses; (2) a parallel multi-arm multi-stage trial and (3) the design used in WIRE, which we call the Multi-Arm Sequential Trial with Efficient Recruitment (MASTER) design. The MASTER design recruits patients to one arm at a time, pausing recruitment to an arm when it has recruited the required number for an interim analysis. We conduct a simulation study to compare how long the three different trial designs take to evaluate a number of new treatment arms. The parallel multi-arm multi-stage and the MASTER design are much more efficient than separate trials. The MASTER design provides extra efficiency when there is endpoint delay, or recruitment is very quick. We recommend the MASTER design as an efficient way of testing multiple promising cancer treatments in non-comparative Phase II trials.
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影响因子：
8.4
作者：
Wason, James M. S.;Mander, Adrian P.;Eisen, Tim G.
通讯作者：
Eisen, Tim G.
影响因子：
2.3
作者：
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作者：
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通讯作者：
Billingham L
DOI：
10.1111/j.1467-9868.2012.01030.x
发表时间：
2013-01-01
影响因子：
5.8
作者：
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通讯作者：
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影响因子：
4.7
作者：
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通讯作者：
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