首页> 外文期刊>Biometrics: Journal of the Biometric Society : An International Society Devoted to the Mathematical and Statistical Aspects of Biology >Inference for the effect of treatment on survival probability in randomized trials with noncompliance and administrative censoring.
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Inference for the effect of treatment on survival probability in randomized trials with noncompliance and administrative censoring.

机译:在不服从和行政检查的随机试验中,推断治疗对生存概率的影响。

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In many clinical studies with a survival outcome, administrative censoring occurs when follow-up ends at a prespecified date and many subjects are still alive. An additional complication in some trials is that there is noncompliance with the assigned treatment. For this setting, we study the estimation of the causal effect of treatment on survival probability up to a given time point among those subjects who would comply with the assignment to both treatment and control. We first discuss the standard instrumental variable (IV) method for survival outcomes and parametric maximum likelihood methods, and then develop an efficient plug-in nonparametric empirical maximum likelihood estimation (PNEMLE) approach. The PNEMLE method does not make any assumptions on outcome distributions, and makes use of the mixture structure in the data to gain efficiency over the standard IV method. Theoretical results of the PNEMLE are derived and the method is illustrated by an analysis of data from a breast cancer screening trial. From our limited mortality analysis with administrative censoring times 10 years into the follow-up, we find a significant benefit of screening is present after 4 years (at the 5% level) and this persists at 10 years follow-up.
机译:在许多具有生存结果的临床研究中,当随访在预定的日期结束并且许多受试者还活着时,就会进行行政检查。在某些试验中,另一个并发症是不符合指定的治疗方法。在这种情况下,我们研究了既要满足治疗又要控制的受试者中,给定时间点治疗对生存概率的因果影响的估计。我们首先讨论用于生存结果的标准工具变量(IV)方法和参数最大似然方法,然后开发一种有效的插件式非参数经验最大似然估计(PNEMLE)方法。 PNEMLE方法不对结果分布进行任何假设,而是利用数据中的混合结构来获得优于标准IV方法的效率。得出PNEMLE的理论结果,并通过对乳腺癌筛查试验数据的分析来说明该方法。从我们有限的死亡率分析(包括随访的10年行政检查时间)中,我们发现筛查在4年后(以5%的水平)存在显着的益处,并且在随访10年后仍然存在。

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