Choice and interpretation of statistical tests used when competing risks are present
Choice and interpretation of statistical tests used when competing risks are present
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DOI：
10.1200/jco.2007.12.9866
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发表时间：
2008-08-20
影响因子：
45.3
通讯作者：
Kocherginsky, Maria N.
中科院分区：
文献类型：
--
作者：
Dignam, James J.;Kocherginsky, Maria N.
在临床癌症研究中，经常会遇到相互竞争的风险。例如，接受手术可切除疾病治疗的个体可能在任何这些事件之前经历切除肿瘤附近的复发、其他部位的转移性复发、第二原发性癌症的发生或非癌症原因导致的死亡。两个量，原因特异性风险函数和累积发生率函数，通常用于按事件类型总结结局。因此，治疗组间事件特异性差异的检验可能基于（a）通过对数秩或相关检验进行的原因特异性危害比较，或（B）通过几种可用检验之一进行的累积发生率函数比较。基于这些不同指标的测试的推断结果对于相同的病因特异性终点可能会有很大差异。根据主要关注的问题，一个或两个指标可能适合考虑。我们提出了模拟研究结果，并讨论了癌症临床试验的例子，以说明这些点，并提供指导分析时，存在竞争的风险。
In clinical cancer research, competing risks are frequently encountered. For example, individuals undergoing treatment for surgically resectable disease may experience recurrence near the removed tumor, metastatic recurrence at other sites, occurrence of second primary cancer, or death resulting from noncancer causes before any of these events. Two quantities, the cause-specific hazard function and the cumulative incidence function, are commonly used to summarize outcomes by event type. Tests for event-specific differences between treatment groups may thus be based on comparison of ( a) cause-specific hazards via a log-rank or related test, or (b) the cumulative incidence functions via one of several available tests. Inferential results for tests based on these different metrics can differ considerably for the same cause-specific end point. Depending on the questions of principal interest, one or both metrics may be appropriate to consider. We present simulation study results and discuss examples from cancer clinical trials to illustrate these points and provide guidance for analysis when competing risks are present.