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

Stat Methods Med Res. 2018 Aug;27(8):2384-2400. doi: 10.1177/0962280216680524. Epub 2016 Dec 29.

Abstract

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.

Keywords: Missing data; censored-by-death; composite endpoints; multiple endpoints; survivor bias; weighted Wilcoxon–Mann–Whitney test.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Brain Ischemia / mortality
  • Brain Ischemia / therapy
  • Humans
  • Mortality*
  • Oxygen Inhalation Therapy
  • Randomized Controlled Trials as Topic*
  • Statistics, Nonparametric*
  • Stroke / mortality
  • Stroke / therapy