Format

Send to

Choose Destination
Lancet Neurol. 2014 Apr;13(4):419-28. doi: 10.1016/S1474-4422(14)70003-1. Epub 2014 Feb 27.

Outcome markers for clinical trials in cerebral amyloid angiopathy.

Author information

1
Stroke Research Center, Massachusetts General Hospital, Boston, MA, USA. Electronic address: sgreenberg@partners.org.
2
Division of Clinical Neurosciences, Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
3
Brain Centre Rudolf Magnus, University Medical Center of Utrecht, Utrecht, Netherlands.
4
Department of Radiology, Leiden University Medical Center, Leiden, Netherlands.
5
Department of Neurology, Universite Lille Nord de France EA 1046, Lille University Hospital, Lille, France.
6
Department of Neurology, Department of Radiology, and Department of Biomedical Engineering, Washington University School of Medicine, St Louis, MO, USA.
7
Department of Neurology, Vall d'Hebron University Hospital and Research Institute, Autonomus University of Barcelona, Barcelona, Spain.
8
Department of Pathology and Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.
9
Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
10
Department of Radiology and Epidemiology, Erasmus University Medical Center, Rotterdam, Netherlands.
11
UCL Institute of Neurology, London, UK.

Abstract

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.

PMID:
24581702
PMCID:
PMC4085787
DOI:
10.1016/S1474-4422(14)70003-1
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for Elsevier Science Icon for PubMed Central
Loading ...
Support Center