Outcome markers for clinical trials in cerebral amyloid angiopathy

Summary Efforts are underway for early-phase trials of candidate treatments for cerebral amyloid angiopathy, an untreatable cause of haemorrhagic stroke and vascular cognitive impairment. A major barrier to these trials is the absence of consensus on measurement of treatment effectiveness. A range o...

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Bibliographic Details
Published in:Lancet neurology 2014-04, Vol.13 (4), p.419-428
Main Authors: Greenberg, Steven M, Prof, Salman, Rustam Al-Shahi, Prof, Biessels, Geert Jan, Prof, van Buchem, Mark, Prof, Cordonnier, Charlotte, Prof, Lee, Jin-Moo, Prof, Montaner, Joan, Prof, Schneider, Julie A, Prof, Smith, Eric E, MD, Vernooij, Meike, MD, Werring, David J, MD
Format: Article
Language:English
Subjects:
Biomarkers
Biomedical research
Brain research
Cerebral Amyloid Angiopathy - pathology
Cerebral Amyloid Angiopathy - therapy
Clinical trials
Disease
Efficiency
Hemorrhage
Hospitals
Humans
Medical imaging
Medical research
Neurology
Stroke
Studies
Treatment Outcome
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Summary:Summary Efforts are underway for early-phase trials of candidate treatments for cerebral amyloid angiopathy, an untreatable cause of haemorrhagic stroke and vascular cognitive impairment. A major barrier to these trials is the absence of consensus on measurement of treatment effectiveness. A range of potential outcome markers for cerebral amyloid angiopathy can be measured against the ideal criteria of being clinically meaningful, closely representative of biological progression, efficient for small or short trials, reliably measurable, and cost effective. In practice, outcomes tend either to have high clinical salience but low statistical efficiency, and thus more applicability for late-phase studies, or greater statistical efficiency but more limited clinical meaning. The most statistically efficient markers might be those that are potentially reversible with treatment, although their clinical significance remains unproven. Many of the candidate outcomes for cerebral amyloid angiopathy trials are probably applicable also to other small-vessel brain diseases.
ISSN:1474-4422
1474-4465
DOI:10.1016/S1474-4422(14)70003-1