On the analysis and interpretation of outcome measures in stroke clinical trials: lessons from the SAINT I study of NXY-059 for acute ischemic stroke.

Abstract:

BACKGROUND AND PURPOSE:A variety of primary end points have been used in acute stroke trials. We focus on the modified Rankin Scale, a reliable and valid ordinal outcome measure that assesses disability on a 7-point scale. METHODS:We provide an abbreviated discussion of analytical methods for ordinal scales, and related effect size measures; we illustrate these methods and their interpretation with outcome data from the SAINT I study of NXY-059 in acute ischemic stroke. RESULTS:The nonparametric Mann-Whitney statistic provides a straightforward method for analysis of the modified Rankin Scale, and incorporates associated measures of effect size. These measures are directly related to the concepts of Number Needed to Treat and Number Needed to Harm. CONCLUSIONS:Our re-examination of the outcome data from the SAINT I study provides little evidence for the purported efficacy of NXY-059. More broadly, analysis and interpretation of ordinal outcome scales based on ascribed numerical values to the steps of the scale should be done cautiously. Statistical treatment of multiple primary outcome measures in acute stroke clinical trials should be established before analysis. Lastly, conflating statistical and clinical significance should be avoided.

journal_name

Stroke

journal_title

Stroke

authors

Koziol JA,Feng AC

doi

10.1161/01.STR.0000241106.81293.2b

subject

Has Abstract

pub_date

2006-10-01 00:00:00

pages

2644-7

issue

10

eissn

0039-2499

issn

1524-4628

pii

01.STR.0000241106.81293.2b

journal_volume

37

pub_type

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