%0 Journal Article
%A Cash, David M
%A Morgan, Katy E
%A O'Connor, Antoinette
%A Veale, Thomas D
%A Malone, Ian B
%A Poole, Teresa
%A Benzinger, Tammie Ls
%A Gordon, Brian A
%A Ibanez, Laura
%A Li, Yan
%A Llibre-Guerra, Jorge J
%A McDade, Eric
%A Wang, Guoqiao
%A Chhatwal, Jasmeer P
%A Day, Gregory S
%A Huey, Edward
%A Jucker, Mathias
%A Levin, Johannes
%A Niimi, Yoshiki
%A Noble, James M
%A Roh, Jee Hoon
%A Sánchez-Valle, Racquel
%A Schofield, Peter R
%A Bateman, Randall J
%A Frost, Chris
%A Fox, Nick C
%T Sample size estimates for biomarker-based outcome measures in clinical trials in autosomal dominant Alzheimer's disease.
%J The journal of prevention of Alzheimer's disease
%V 12
%N 6
%@ 2274-5807
%C Cham
%I Springer International Publishing
%M DZNE-2025-00781
%P 100133
%D 2025
%X Alzheimer disease (AD)-modifying therapies are approved for treatment of early-symptomatic AD. Autosomal dominant AD (ADAD) provides a unique opportunity to test therapies in presymptomatic individuals.Using data from the Dominantly Inherited Alzheimer Network (DIAN), sample sizes for clinical trials were estimated for various cognitive, imaging, and CSF outcomes. Sample sizes were computed for detecting a reduction of either absolute levels of AD-related pathology (amyloid, tau) or change over time in neurodegeneration (atrophy, hypometabolism, cognitive change).Biomarkers measuring amyloid and tau pathology had required sample sizes below 200 participants per arm (examples CSF Aβ42/40: 47[95 
%K Humans
%K Alzheimer Disease: genetics
%K Alzheimer Disease: cerebrospinal fluid
%K Alzheimer Disease: diagnosis
%K Alzheimer Disease: diagnostic imaging
%K Alzheimer Disease: pathology
%K Biomarkers: cerebrospinal fluid
%K Amyloid beta-Peptides: cerebrospinal fluid
%K Sample Size
%K tau Proteins: cerebrospinal fluid
%K Male
%K Female
%K Clinical Trials as Topic
%K Outcome Assessment, Health Care
%K Magnetic Resonance Imaging
%K Positron-Emission Tomography
%K Brain: pathology
%K Brain: diagnostic imaging
%K Middle Aged
%K Alzheimer's disease (Other)
%K Autosomal dominant (Other)
%K CSF (Other)
%K Clinical trials (Other)
%K Linear mixed effects models (Other)
%K Longitudinal (Other)
%K MRI (Other)
%K PET (Other)
%K Sample size (Other)
%K ß-amyloid (Other)
%K Biomarkers (NLM Chemicals)
%K Amyloid beta-Peptides (NLM Chemicals)
%K tau Proteins (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:40118731
%R 10.1016/j.tjpad.2025.100133
%U https://pub.dzne.de/record/279454