Assessing risk for preclinical β-amyloid pathology with APOE, cognitive, and demographic information

Research output: Contribution to journalArticle


Introduction Clinical trials in Alzheimer's disease are aimed at early stages of disease, including preclinical Alzheimer's disease. The high cost and time required to screen large numbers of participants for Aβ pathology impede the development of novel drugs. This study's objective was to evaluate the extent to which inexpensive and easily obtainable information can reduce the number of screen failures by increasing the proportion of Aβ+ participants identified for screening. Methods We used random forest models to evaluate the positive predictive value of demographics, APOE, and longitudinal cognitive rates in the prediction of amyloid pathology, measured by florbetapir PET or cerebrospinal fluid. Results Predicting Aβ positivity with demographic, APOE, and cognitive information yielded a positive predictive value estimate of 0.65 (95% CI, 0.50–0.96), nearly a 60% increase over the reference Aβ+ prevalence in the cohort of 0.41. Conclusions By incorporating this procedure, clinical trial screening costs of 7500 USD per participant may be reduced by nearly 7 million USD total.


External organisations
  • Skåne University Hospital
  • University of California System
  • San Francisco Veterans Administration Medical Center
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Neurology


  • Amyloid, APOE, Clinical trials, Cognition, Preclinical Alzheimer's
Original languageEnglish
Pages (from-to)76-84
Number of pages9
JournalAlzheimer's and Dementia: Diagnosis, Assessment and Disease Monitoring
Publication statusPublished - 2016
Publication categoryResearch