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Neurodegener Dis. 2014;13(2-3):197-9. doi: 10.1159/000353748. Epub 2013 Aug 7.

How can we improve transfer of outcomes from randomized clinical trials to clinical practice with disease-modifying drugs in Alzheimer's disease?

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McGill Center for Studies in Aging, Douglas Mental Health Research Institute, Montreal, Que., Canada.



Randomized clinical trials (RCTs) for putative disease-modifying drugs in Alzheimer's disease (AD) are using cognitive outcomes, such as the Alzheimer's Disease Assessment Scale--cognitive subscale, activities of daily living scales, such as the Alzheimer's Disease Cooperative Study Activities of Daily Living, and time from mild cognitive impairment to AD dementia.


It was the aim of this study to build clinically relevant outcomes for future use in clinical practice into RCT designs and help third-party payers to measure benefit.


We used a literature review for analysis.


The Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) appears to be the most reliable primary outcome for RCT at different stages of AD, with the Relevant Outcome Scale for Alzheimer's Disease (ROSA) as a suitable alternative. The importance of current AD biomarkers vis-à- vis determination of efficacy of disease-modifying drugs has yet to be established; however, it is likely that at least one amyloid-specific test will be required prior to treatment with a drug acting predominantly on β-amyloid (Aβ42). Furthermore, serial MRI may be required to monitor adverse side effects associated with such drugs.


Global clinical scales such as CDR-SB and ROSA should be considered for use with treatments aiming at slowing disease progression.

[Indexed for MEDLINE]

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