Unlocking Cognitive Analysis Potential in Alzheimer's Disease Clinical Trials: Investigating Hierarchical Linear Models for Analyzing Novel Measurement Burst Design Data

Stat Med. 2024 Dec 30;43(30):5898-5910. doi: 10.1002/sim.10292. Epub 2024 Nov 25.

Abstract

Measurement burst designs typically administer brief cognitive tests four times per day for 1 week, resulting in a maximum of 28 data points per week per test for every 6 months. In Alzheimer's disease clinical trials, utilizing measurement burst designs holds great promise for boosting statistical power by collecting huge amount of data. However, appropriate methods for analyzing these complex datasets are not well investigated. Furthermore, the large amount of burst design data also poses tremendous challenges for traditional computational procedures such as SAS mixed or Nlmixed. We propose to analyze burst design data using novel hierarchical linear mixed effects models or hierarchical mixed models for repeated measures. Through simulations and real-world data applications using the novel SAS procedure Hpmixed, we demonstrate these hierarchical models' efficiency over traditional models. Our sample simulation and analysis code can serve as a catalyst to facilitate the methodology development for burst design data.

Keywords: Alzheimer's disease; SAS; hierarchical linear mixed effects model; hierarchical mixed models for repeated measures; measurement burst design data.

MeSH terms

  • Alzheimer Disease*
  • Clinical Trials as Topic
  • Cognition
  • Computer Simulation*
  • Data Interpretation, Statistical
  • Humans
  • Linear Models
  • Research Design