Com-Nougue C, Rodary C, Patte C
Université Paris VI, Unité 351 de l'Institut National de la Santé et de la Recherche Médicale, Institut Gustave Roussy, Villejuif, France.
Stat Med. 1993 Jul 30;12(14):1353-64. doi: 10.1002/sim.4780121407.
Interest in equivalence trials has been increasing for many years, though the methodology which has been developed for such trials is mainly for uncensored data. In cancer research we are more often concerned with survival. In an efficacy trial, the null hypothesis specifies equality of the two survival distributions, but in an equivalence trial, a null hypothesis of inequivalence H0 has to be tested. The usual logrank test has to be modified to test whether the true value r of the ratio of hazard rates in two treatment groups is at least equal to a limit value r0. If prognostic factors have to be taken into account, the Cox model provides tests of Ho, and a useful confidence interval for the adjusted relative derived from the regression parameter for the treatment indicator. An equivalence trial of maintenance therapy was carried out in children with B non-Hodgkin lymphoma, and serves as an illustration.
多年来，人们对等效性试验的兴趣一直在增加，尽管为此类试验开发的方法主要适用于未删失数据。在癌症研究中，我们更常关注生存率。在疗效试验中，原假设规定两种生存分布相等，但在等效性试验中，必须检验不等效的原假设H0。通常的对数秩检验必须进行修改，以检验两个治疗组中风险率之比的真实值r是否至少等于一个极限值r0。如果必须考虑预后因素，Cox模型可提供对H0的检验，以及从治疗指标的回归参数得出的调整后相对值的有用置信区间。对B非霍奇金淋巴瘤患儿进行了维持治疗的等效性试验，并作为一个示例。
