Outcome analysis in clinical trial design for acute stroke: physicians' attitudes and choices
Department of Neurology
*Attitude of Health Personnel; *Choice Behavior; *Clinical Trials as Topic; Comprehension; Cross-Sectional Studies; Data Interpretation, Statistical; Endpoint Determination; Female; *Health Knowledge, Attitudes, Practice; Hospitals, University; Humans; Internet; Male; Neuroprotective Agents; Patient Selection; Severity of Illness Index; Stroke; Treatment Outcome; United States
Neurology | Neuroscience and Neurobiology
BACKGROUND: Thrombolysis remains the only proven therapy to benefit acute ischemic stroke (AIS) patients. Recent studies have introduced more sensitive outcome measures such as the shift analysis to detect a treatment effect in AIS trials and are also including imaging as a surrogate of injury.
METHODS: We conducted a cross-sectional, internet-based survey of academic neurologists regarding their attitudes, choices and understanding of various outcome measures in clinical trial design for AIS. The survey population consisted of neurologists who specialize in the care of stroke patients and are on faculty at university-affiliated hospitals in the USA.
RESULTS: 152 of 300 neurologists completed the survey. There were 79% men and 21% women. Among commonly used outcome scales in acute stroke, the most frequent ones selected for use as trial primary endpoints were the global statistic (59%), modified Rankin scale (mRS) (52%), and NIHSS (30%). When given choices about which outcome on the mRS would justify a therapeutic intervention, 54% chose a shift analysis of change in the distribution of outcomes and 39% chose a dichotomized outcome (mRS
CONCLUSION: The majority of respondents accepted an analysis of the entire distribution of the mRS scores as an appropriate endpoint analytic technique in AIS trials and did not require the traditional dichotomized outcome to demonstrate a treatment effect; however, a better understanding of the shift strategy is needed. Our data also support the importance of incorporating mismatch imaging into future neuroprotection trials.
DOI of Published Version
Cerebrovasc Dis. 2008;26(2):156-62. Epub 2008 Jun 17. Link to article on publisher's site
Cerebrovascular diseases (Basel, Switzerland)
Savitz SI, Benatar M, Saver JL, Fisher M. (2008). Outcome analysis in clinical trial design for acute stroke: physicians' attitudes and choices. Neurology Publications. https://doi.org/10.1159/000139663. Retrieved from https://escholarship.umassmed.edu/neuro_pp/366