Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials

Background and Purpose—Vascular prevention trials mostly count “yes/no” (binary) outcome events, eg, stroke/no stroke. Analysis of ordered categorical vascular events (eg, fatal stroke/nonfatal stroke/no stroke) is clinically relevant and could be more powerful statistically. Although this is not...

Full description

Bibliographic Details
Main Authors: Bath, Philip M.W., Geeganage, Chamila, Gray, Laura J., Collier, T, Pocock, S
Format: Article
Published: American Heart Association 2008
Online Access:https://eprints.nottingham.ac.uk/961/
_version_ 1848790511558590464
author Bath, Philip M.W.
Geeganage, Chamila
Gray, Laura J.
Collier, T
Pocock, S
author_facet Bath, Philip M.W.
Geeganage, Chamila
Gray, Laura J.
Collier, T
Pocock, S
author_sort Bath, Philip M.W.
building Nottingham Research Data Repository
collection Online Access
description Background and Purpose—Vascular prevention trials mostly count “yes/no” (binary) outcome events, eg, stroke/no stroke. Analysis of ordered categorical vascular events (eg, fatal stroke/nonfatal stroke/no stroke) is clinically relevant and could be more powerful statistically. Although this is not a novel idea in the statistical community, ordinal outcomes have not been applied to stroke prevention trials in the past. Methods—Summary data on stroke, myocardial infarction, combined vascular events, and bleeding were obtained by treatment group from published vascular prevention trials. Data were analyzed using 10 statistical approaches which allow comparison of 2 ordinal or binary treatment groups. The results for each statistical test for each trial were then compared using Friedman 2-way analysis of variance with multiple comparison procedures. Results—Across 85 trials (335 305 subjects) the test results differed substantially so that approaches which used the ordinal nature of stroke events (fatal/nonfatal/no stroke) were more efficient than those which combined the data to form 2 groups (P0.0001). The most efficient tests were bootstrapping the difference in mean rank, Mann–Whitney U test, and ordinal logistic regression; 4- and 5-level data were more efficient still. Similar findings were obtained for myocardial infarction, combined vascular outcomes, and bleeding. The findings were consistent across different types, designs and sizes of trial, and for the different types of intervention. Conclusions—When analyzing vascular events from prevention trials, statistical tests which use ordered categorical data are more efficient and are more likely to yield reliable results than binary tests. This approach gives additional information on treatment effects by severity of event and will allow trials to be smaller. (Stroke. 2008;39:000-000.)
first_indexed 2025-11-14T18:13:47Z
format Article
id nottingham-961
institution University of Nottingham Malaysia Campus
institution_category Local University
last_indexed 2025-11-14T18:13:47Z
publishDate 2008
publisher American Heart Association
recordtype eprints
repository_type Digital Repository
spelling nottingham-9612020-05-04T16:27:43Z https://eprints.nottingham.ac.uk/961/ Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials Bath, Philip M.W. Geeganage, Chamila Gray, Laura J. Collier, T Pocock, S Background and Purpose—Vascular prevention trials mostly count “yes/no” (binary) outcome events, eg, stroke/no stroke. Analysis of ordered categorical vascular events (eg, fatal stroke/nonfatal stroke/no stroke) is clinically relevant and could be more powerful statistically. Although this is not a novel idea in the statistical community, ordinal outcomes have not been applied to stroke prevention trials in the past. Methods—Summary data on stroke, myocardial infarction, combined vascular events, and bleeding were obtained by treatment group from published vascular prevention trials. Data were analyzed using 10 statistical approaches which allow comparison of 2 ordinal or binary treatment groups. The results for each statistical test for each trial were then compared using Friedman 2-way analysis of variance with multiple comparison procedures. Results—Across 85 trials (335 305 subjects) the test results differed substantially so that approaches which used the ordinal nature of stroke events (fatal/nonfatal/no stroke) were more efficient than those which combined the data to form 2 groups (P0.0001). The most efficient tests were bootstrapping the difference in mean rank, Mann–Whitney U test, and ordinal logistic regression; 4- and 5-level data were more efficient still. Similar findings were obtained for myocardial infarction, combined vascular outcomes, and bleeding. The findings were consistent across different types, designs and sizes of trial, and for the different types of intervention. Conclusions—When analyzing vascular events from prevention trials, statistical tests which use ordered categorical data are more efficient and are more likely to yield reliable results than binary tests. This approach gives additional information on treatment effects by severity of event and will allow trials to be smaller. (Stroke. 2008;39:000-000.) American Heart Association 2008-07-31 Article PeerReviewed Bath, Philip M.W., Geeganage, Chamila, Gray, Laura J., Collier, T and Pocock, S (2008) Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials. Stroke, 39 (10). pp. 2817-2823. ISSN 1524-4628 http://stroke.ahajournals.org/content/39/10/2817 doi:10.1161/STROKEAHA.107.509893 doi:10.1161/STROKEAHA.107.509893
spellingShingle Bath, Philip M.W.
Geeganage, Chamila
Gray, Laura J.
Collier, T
Pocock, S
Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title_full Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title_fullStr Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title_full_unstemmed Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title_short Use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
title_sort use of ordinal outcomes in vascular prevention trials: comparison with binary outcomes in published trials
url https://eprints.nottingham.ac.uk/961/
https://eprints.nottingham.ac.uk/961/
https://eprints.nottingham.ac.uk/961/