OPTIMAL 2-STAGE SCREENING DESIGNS FOR SURVIVAL COMPARISONS
OPTIMAL 2-STAGE SCREENING DESIGNS FOR SURVIVAL COMPARISONS
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DOI：
10.2307/2336988
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发表时间：
1990-09-01
期刊：
影响因子：
2.7
通讯作者：
THERNEAU, TM
中科院分区：
文献类型：
--
作者：
SCHAID, DJ;WIEAND, S;THERNEAU, TM
提出了一种两阶段设计，能够在第一阶段筛选几种新的实验性治疗方法，以提高存活率，而不是标准方案。只有有希望的治疗才会进入第二阶段进行最终评估。我们的程序是，在特定的阿尔法错误、功率和停止规则的限制下，最大限度地减少新方案预计累积的患者数量，这些新方案没有提供比标准方案更好的生存益处，而停止规则允许指定风险比的大小，这将保证在第一阶段之后进行应计。我们通过将我们的设计与各种情况下的单阶段设计进行比较来探索患者应计费用的节省。我们的设计可能会在风险比率相对于患者应收率较大的情况下提供实质性的节省，这种情况在晚期癌症的临床试验中经常遇到。虽然计算是基于渐近结果的，但模拟验证了这些近似对于大多数感兴趣的试验是足够的。
A two-stage design is presented which enables the screening, at the first stage, of several new experimental treatments for survival improvement over a standard regimen. Only promising treatments are carried forward to the second stage for definitive evaluation. Our procedure is to minimize the number of patients expected to be accrued to new regimens which do not offer a survival benefit over the standard regimen, subject to constraints of specified alpha-error, power, and a stopping rule which allows specification of the magnitude of the hazards ratio which would warrant accrual beyond the first stage. We explore the savings in patient accrual by comparing our design with a single-stage design for a variety of situations. Our design may offer a substantial saving when the hazard rate is large relative to the patient accrual rate, a situation often encountered in clinical trials in advanced cancer. Although computations are based on asymptotic results, simulations verify that the approximations are adequate for most trials of interest.