Competing risks data are common in medical studies, and the sub-distribution hazard (SDH) ratio is considered an appropriate measure. However, because the limitations of hazard itself are not easy to interpret clinically and because the SDH ratio is valid only under the proportional SDH assumption, this article introduced an alternative index under competing risks, named restricted mean time lost (RMTL). Several test procedures were also constructed based on RMTL. First, we introduced the definition and estimation of RMTL based on Aalen-Johansen cumulative incidence functions. Then, we considered several combined tests based on the SDH and the RMTL difference (RMTLd). The statistical properties of the methods are evaluated using simulations and are applied to two examples. The type I errors of combined tests are close to the nominal level. All combined tests show acceptable power in all situations. In conclusion, RMTL can meaningfully summarize treatment effects for clinical decision making, and three combined tests have robust power under various conditions, which can be considered for statistical inference in real data analysis.
翻译:相竞风险数据在医学研究中很常见,分分配危险比率被认为是一项适当的措施,然而,由于危险本身的局限性本身不容易临床解释,而且由于SDH比率仅在比例的SDH假设下有效,本条引入了竞争性风险的替代指数,称为限制平均损失时间(RMTL),还根据RMTL制定了若干测试程序。首先,我们引入了基于Aalen-Johansen累积发生率功能的RMTL定义和估计。然后,我们考虑了基于SDH和RMTL差异(RMTLd)的几项综合测试。这些方法的统计特性通过模拟来评估,并应用到两个例子。综合测试的第一类错误接近于名义水平。所有综合测试都表明在所有情况下都具有可接受的能力。最后,RMTL可以有意义地总结临床决策的治疗效果,而三种合并测试在各种条件下都具有强大的能力,可以考虑在真实数据分析中进行统计推断。