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

Abstract

This article analyzes various inference techniques for the CC-index p=1/2(P(T1>T2)+P(T1T2))p = 1/2 (P(T_1 > T_2) + P(T_1 \geq T_2)) and the win ratio w=p/(1p)w = p / (1-p) for possibly discretely distributed, independent survival times T1T_1 and T2T_2. While observation of T1T_1 and T2T_2 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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