Outcome markers for clinical trials in cerebral amyloid angiopathy

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• 作者：Prof Steven M Greenberg ; MD^a ; ^{sgreenberg@partners.org" class="auth_mail} ; Prof Rustam Al-Shahi Salman ; PhD^b ; Prof Geert Jan Biessels ; MD^c ; Prof Mark van Buchem ; MD^d ; Prof Charlotte Cordonnier ; MD^e ; Prof Jin-Moo Lee ; MD^f ; Prof Joan Montaner ; MD^g ; Prof Julie A Schneider ; MD^h ; Eric E Smith ; MDⁱ ; Meike Vernooij ; MD^j ; David J Werring ; MD^k
• 刊名：Lancet Neurology
• 出版年：April, 2014
• 年：2014
• 卷：13
• 期：4
• 页码：419-428
• 全文大小：769 K

文摘

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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.