An optimal Wilcoxon-Mann-Whitney test of mortality and a continuous outcome

作者：Matsouaka Roland A.^*; Singhal Aneesh B.; Betensky Rebecca A.

来源：Statistical Methods in Medical Research, 2018, 27(8): 2384-2400.

DOI：10.1177/0962280216680524

• Missing data
• survivor bias
• multiple endpoints
• weighted Wilcoxon-Mann-Whitney test
• censored-by-death
• composite endpoints

We consider a two-group randomized clinical trial, where mortality affects the assessment of a follow-up continuous outcome. Using the worst-rank composite endpoint, we develop a weighted Wilcoxon-Mann-Whitney test statistic to analyze the data. We determine the optimal weights for the Wilcoxon-Mann-Whitney test statistic that maximize its power. We derive a formula for its power and demonstrate its accuracy in simulations. Finally, we apply the method to data from an acute ischemic stroke clinical trial of normobaric oxygen therapy.

• 出版日期2018-8