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Resampling-based Confidence Intervals and Tests for the Concordance Index and the Win Ratio

16 May 2016
D. Dobler
Markus Pauly
ArXiv (abs)PDFHTML
Abstract

This article analyzes various inference techniques for the CCC-index p=1/2(P(T1>T2)+P(T1≥T2))p = 1/2 (P(T_1 > T_2) + P(T_1 \geq T_2))p=1/2(P(T1​>T2​)+P(T1​≥T2​)) and the win ratio w=p/(1−p)w = p / (1-p)w=p/(1−p) for possibly discretely distributed, independent survival times T1T_1T1​ and T2T_2T2​. While observation of T1T_1T1​ and T2T_2T2​ may be right-censored and are thus dealt with by the Kaplan-Meier estimator, observations larger than the end-of-study time are also reasonably accounted for. An appropiate handling of ties requires normalized versions of Kaplan-Meier and variance estimators. Asymptotically exact inference procedures based on standard normal quantiles are compared to their bootstrap- and permutation-based versions. A simulation study presents a robust superiority of permutation-based procedures over the non-resampling counterparts −-− even for small, unequally sized samples, strong censoring and under different sample distributions.

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