Trial designs and outcomes to monitor novel therapeutics in Alzheimer's disease

Abstract

The various etiological hypotheses for Alzheimer's disease (AD) need to be tested in patients using designs and outcomes that are appropriate. This chapter reviews the principles of symptomatic treatment versus disease modification, the natural history of AD, and designs to slow down its progression. It should be noted that the diagnosis of AD implies first a diagnosis of dementia, followed by an assessment of its etiology. The accuracy of the clinical diagnosis of AD is in the order of 85% once dementia is clinically detected, but less than 50% in the predementia stage of amnestic mild cognitive impairment (aMCI) using clinicopathological correlations (Petersen et al., 2006).