title: An optimal Wilcoxon-Mann-Whitney test of mortality and a continuous outcome.
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.
authors.affiliation: 1 Department of Biostatistics and Bioinformatics, Duke University, Durham, NC, USA.|2 Duke Clinical Research Institute, Duke University, Durham, NC, USA.
authors.forename: Roland A
authors.initials: RA
authors.lastname: Matsouaka
authors.affiliation: 3 Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
authors.forename: Aneesh B
authors.initials: AB
authors.lastname: Singhal
authors.affiliation: 4 Department of Biostatistics, Harvard T.H. Chan School of Public Health.|5 Harvard NeuroDiscovery Center, Harvard Medical School.
authors.forename: Rebecca A
authors.initials: RA
authors.lastname: Betensky
mesh_terms: D002545:Brain Ischemia / Q000401:mortality / Q000628:therapy; D006801:Humans; D009026:Mortality; D010102:Oxygen Inhalation Therapy; D016032:Randomized Controlled Trials as Topic; D018709:Statistics, Nonparametric; D020521:Stroke / Q000401:mortality / Q000628:therapy
keywords: Missing data; censored-by-death; composite endpoints; multiple endpoints; survivor bias; weighted Wilcoxon–Mann–Whitney test