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Testing and interval estimation for two-sample survival comparisons with small sample sizes and unequal censoring

机译:小样本量和不平等审查的两样本生存比较的测试和区间估计

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摘要

While the commonly used log-rank test for survival times between 2 groups enjoys many desirable properties, sometimes the log-rank test and its related linear rank tests perform poorly when sample sizes are small. Similar concerns apply to interval estimates for treatment differences in this setting, though their properties are less well known. Standard permutation tests are one option, but these are not in general valid when the underlying censoring distributions in the comparison groups are unequal. We develop 2 methods for testing and interval estimation, for use with small samples and possibly unequal censoring, based on first imputing survival and censoring times and then applying permutation methods. One provides a heuristic justification for the approach proposed recently by Heinze and others (2003, Exact log-rank tests for unequal follow-up. Biometrics 59, 1151–1157). Simulation studies show that the proposed methods have good Type I error and power properties. For accelerated failure time models, compared to the asymptotic methods of Jin and others (2003, Rank-based inference for the accelerated failure time model. Biometrika 90, 341–353), the proposed methods yield confidence intervals with better coverage probabilities in small-sample settings and similar efficiency when sample sizes are large. The proposed methods are illustrated with data from a cancer study and an AIDS clinical trial.
机译:尽管常用的2组之间生存时间的对数秩检验具有许多理想的属性,但有时,当样本量较小时,对数秩检验及其相关的线性秩检验效果较差。尽管对它们的属性了解较少,但类似的担忧也适用于这种情况下治疗差异的区间估计。标准排列测试是一种选择,但是当比较组中的基础检查分布不相等时,这些通常无效。我们首先根据估算的生存时间和审查时间,然后应用置换方法,开发了两种用于测试和区间估计的方法,用于小样本和可能不平等的审查。一种方法为Heinze等人最近提出的方法提供了一种启发式的理由(2003年,对不平等的随访进行精确的对数秩检验。Biometrics59,1151–1157)。仿真研究表明,该方法具有良好的I类误差和功率特性。对于加速故障时间模型,与Jin等人的渐进方法(2003年,基于加速故障时间模型的基于秩的推断。Biometrika90,341–353)相比,所提出的方法在置信区间内具有较小的覆盖概率。样本量大时,样本设置和类似效率。癌症研究和艾滋病临床试验的数据说明了所提出的方法。

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