iStats Inc ., Long Island City, New York, USA.
Department of Biostatistics and Medical Informatics, University of Wisconsin , Madison, Wisconsin, USA.
J Biopharm Stat. 2020 Sep 2;30(5):882-899. doi: 10.1080/10543406.2020.1757692. Epub 2020 Jun 17.
The win ratio method has received much attention in methodological research, ad hoc analyses, and designs of prospective studies. As the primary analysis, it supported the approval of tafamidis for the treatment of cardiomyopathy to reduce cardiovascular mortality and cardiovascular-related hospitalization. However, its dependence on censoring is a potential shortcoming. In this article, we propose the inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic (i.e., the IPCW-adjusted win ratio statistic) to overcome censoring issues. We consider independent censoring, common censoring across endpoints, and right censoring. We develop an asymptotic variance estimator for the logarithm of the IPCW-adjusted win ratio statistic and evaluate it via simulation. Our simulation studies show that, as the amount of censoring increases, the unadjusted win proportions may decrease greatly. Consequently, the bias of the unadjusted win ratio estimate may increase greatly, producing either an overestimate or an underestimate. We demonstrate theoretically and through simulation that the IPCW-adjusted win ratio statistic gives an unbiased estimate of treatment effect.
优势比法在方法学研究、专门分析和前瞻性研究设计中受到了广泛关注。作为主要分析方法,它支持批准塔法米地斯用于治疗心肌病,以降低心血管死亡率和与心血管相关的住院率。然而,它对删失的依赖性是一个潜在的缺点。在本文中,我们提出了逆概率删失加权(Inverse-Probability-of-Censoring Weighting,IPCW)调整的优势比统计量(即 IPCW 调整的优势比统计量)来克服删失问题。我们考虑了独立删失、终点间的共同删失和右删失。我们为 IPCW 调整的优势比统计量的对数开发了一个渐近方差估计量,并通过模拟进行了评估。我们的模拟研究表明,随着删失量的增加,未经调整的优势比例可能会大幅下降。因此,未经调整的优势比估计的偏差可能会大幅增加,导致高估或低估。我们从理论上和模拟中证明,IPCW 调整的优势比统计量给出了治疗效果的无偏估计。