A novel cognitive disease progression model for clinical trials in autosomal-dominant Alzheimer's disease.

ABSTRACT: Clinical trial outcomes for Alzheimer's disease are typically analyzed by using the mixed model for repeated measures (MMRM) or similar models that compare an efficacy scale change from baseline between treatment arms with or without participants' disease stage as a covariate. The MMRM focuses on a single-point fixed follow-up duration regardless of the exposure for each participant. In contrast to these typical models, we have developed a novel semiparametric cognitive disease progression model (DPM) for autosomal dominant Alzheimer's disease based on the Dominantly Inherited Alzheimer Network (DIAN) observational study. This model includes 3 novel features, in which the DPM (1) aligns and compares participants by disease stage, (2) uses a proportional treatment effect similar to the conc

SUBMITTER: Wang G 

PROVIDER: S-EPMC6105413 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature