Design and sample size considerations for Alzheimer's disease prevention trials using multistate models.

ABSTRACT: BACKGROUND/AIMS:Clinical trials for Alzheimer's disease have been aimed primarily at persons who have cognitive symptoms at enrollment. However, researchers are now recognizing that the pathophysiological process of Alzheimer's disease begins years, if not decades, prior to the onset of clinical symptoms. Successful intervention may require intervening early in the disease process. Critical issues arise in designing clinical trials for primary and secondary prevention of Alzheimer's disease including determination of sample sizes and follow-up duration. We address a number of these issues through application of a unifying multistate model for the preclinical course of Alzheimer's disease. A multistate model allows us to specify at which points during the long disease process the interventi

SUBMITTER: Brookmeyer R 

PROVIDER: S-EPMC6442939 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature