TY  - JOUR
AU  - Cash, David M
AU  - Morgan, Katy E
AU  - O'Connor, Antoinette
AU  - Veale, Thomas D
AU  - Malone, Ian B
AU  - Poole, Teresa
AU  - Benzinger, Tammie Ls
AU  - Gordon, Brian A
AU  - Ibanez, Laura
AU  - Li, Yan
AU  - Llibre-Guerra, Jorge J
AU  - McDade, Eric
AU  - Wang, Guoqiao
AU  - Chhatwal, Jasmeer P
AU  - Day, Gregory S
AU  - Huey, Edward
AU  - Jucker, Mathias
AU  - Levin, Johannes
AU  - Niimi, Yoshiki
AU  - Noble, James M
AU  - Roh, Jee Hoon
AU  - Sánchez-Valle, Racquel
AU  - Schofield, Peter R
AU  - Bateman, Randall J
AU  - Frost, Chris
AU  - Fox, Nick C
TI  - Sample size estimates for biomarker-based outcome measures in clinical trials in autosomal dominant Alzheimer's disease.
JO  - The journal of prevention of Alzheimer's disease
VL  - 12
IS  - 6
SN  - 2274-5807
CY  - Cham
PB  - Springer International Publishing
M1  - DZNE-2025-00781
SP  - 100133
PY  - 2025
AB  - 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 
KW  - Humans
KW  - Alzheimer Disease: genetics
KW  - Alzheimer Disease: cerebrospinal fluid
KW  - Alzheimer Disease: diagnosis
KW  - Alzheimer Disease: diagnostic imaging
KW  - Alzheimer Disease: pathology
KW  - Biomarkers: cerebrospinal fluid
KW  - Amyloid beta-Peptides: cerebrospinal fluid
KW  - Sample Size
KW  - tau Proteins: cerebrospinal fluid
KW  - Male
KW  - Female
KW  - Clinical Trials as Topic
KW  - Outcome Assessment, Health Care
KW  - Magnetic Resonance Imaging
KW  - Positron-Emission Tomography
KW  - Brain: pathology
KW  - Brain: diagnostic imaging
KW  - Middle Aged
KW  - Alzheimer's disease (Other)
KW  - Autosomal dominant (Other)
KW  - CSF (Other)
KW  - Clinical trials (Other)
KW  - Linear mixed effects models (Other)
KW  - Longitudinal (Other)
KW  - MRI (Other)
KW  - PET (Other)
KW  - Sample size (Other)
KW  - ß-amyloid (Other)
KW  - Biomarkers (NLM Chemicals)
KW  - Amyloid beta-Peptides (NLM Chemicals)
KW  - tau Proteins (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:40118731
DO  - DOI:10.1016/j.tjpad.2025.100133
UR  - https://pub.dzne.de/record/279454
ER  -