Outcome Measure and Efficacy Analysis in Stroke Clinical Trials

J Korean Neurol Assoc > Volume 24(5); 2006 > Article

Journal of the Korean Neurological Association 2006;24(5): 411-420.

뇌졸중 임상시험에서 치료결과의 측정 및 효과 판정

인제대학교 의과대학 신경과학교실, 임상연구센터

Outcome Measure and Efficacy Analysis in Stroke Clinical Trials

Keun-Sik Hong

Department of Neurology, Clinical Research Center, Inje University College of Medicine, Ilsan, Korea

Abstract

In clinical trials, treatment response should be assessed with appropriate outcome measure and analytic methods. Selection of outcome measure is dependent on the characteristics of subjects and given therapy. For stroke prevention trials, the usual outcome measure is the composite of clinical events such as non-fatal stroke, non-fatal myocardial infarction, or vascular death. On the other hand, for acute stroke trials, stroke severity and functional outcome are essential outcome measures. Neuroimaging is an emerging tool of outcome measure as well as a means of optimizing patient selection in stroke trials. For the analysis of therapeutic efficacy, measured outcomes were usually dichotomized to “good” or “bad” according to arbitrary criteria. Although this approach makes the result of clinical trials easy to interpret, it may have the great chance of missing the clinically important, but modest therapeutic efficacy. To overcome this disadvantage, new analytic strategies such as sliding dichotomy and shift analysis have been proposed. Understanding of outcome measures and analytic methods is important to interpret or design the stroke clinical trials. KeyWords:Stroke, Clinical trial, Outcome measure, Analysis