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

TitleHow can we improve transfer of outcomes from randomized clinical trials to clinical practice with disease-modifying drugs in Alzheimer's disease?
Publication TypeJournal Article
Year of Publication2014
AuthorsGauthier S, Leuzy A, Rosa-Neto P
JournalNeurodegener Dis
Volume13
Issue2-3
Pagination197-9
Date Published2014
ISSN1660-2862
KeywordsAlzheimer Disease, Humans, Neuroprotective Agents, Outcome Assessment (Health Care), Randomized Controlled Trials as Topic, Severity of Illness Index, Translational Medical Research
Abstract

BACKGROUND: 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.OBJECTIVE: 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.METHODS: We used a literature review for analysis.RESULTS: 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.CONCLUSIONS: Global clinical scales such as CDR-SB and ROSA should be considered for use with treatments aiming at slowing disease progression.

DOI10.1159/000353748
Alternate JournalNeurodegener Dis
PubMed ID23942173


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