TY  - JOUR
AU  - Quattrone, Andrea
AU  - Franzmeier, Nicolai
AU  - Huppertz, Hans-Jürgen
AU  - Seneca, Nicholas
AU  - Petzold, Gabor C
AU  - Spottke, Annika
AU  - Levin, Johannes
AU  - Prudlo, Johannes
AU  - Düzel, Emrah
AU  - Höglinger, Günter U
TI  - Brain Atrophy Does Not Predict Clinical Progression in Progressive Supranuclear Palsy.
JO  - Movement disorders
VL  - 40
IS  - 11
SN  - 0885-3185
CY  - New York, NY
PB  - Wiley
M1  - DZNE-2025-01324
SP  - 2517 - 2530
PY  - 2025
AB  - Clinical progression rate is the typical primary endpoint measure in progressive supranuclear palsy (PSP) clinical trials.This longitudinal multicohort study investigated whether baseline clinical severity and regional brain atrophy could predict clinical progression in PSP-Richardson's syndrome (PSP-RS).PSP-RS patients (n = 309) from the placebo arms of clinical trials (NCT03068468, NCT01110720, NCT02985879, NCT01049399) and DescribePSP cohort were included. We investigated associations of baseline clinical and volumetric magnetic resonance imaging (MRI) data with 1-year longitudinal PSP rating scale (PSPRS) change. Machine learning (ML) models were tested to predict individual clinical trajectories.PSP-RS patients showed a mean PSPRS score increase of 10.3 points/yr. The frontal lobe volume showed the strongest association with subsequent clinical progression (β: -0.34, P < 0.001). However, ML models did not accurately predict individual progression rates (R2 <0.15).Baseline clinical severity and brain atrophy could not predict individual clinical progression, suggesting no need for MRI-based stratification of patients in future PSP trials. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
KW  - Humans
KW  - Supranuclear Palsy, Progressive: pathology
KW  - Supranuclear Palsy, Progressive: diagnostic imaging
KW  - Disease Progression
KW  - Atrophy: pathology
KW  - Male
KW  - Female
KW  - Aged
KW  - Magnetic Resonance Imaging
KW  - Brain: pathology
KW  - Brain: diagnostic imaging
KW  - Middle Aged
KW  - Longitudinal Studies
KW  - Severity of Illness Index
KW  - Machine Learning
KW  - atlas‐based volumetry (Other)
KW  - clinical trials (Other)
KW  - outcome (Other)
KW  - progression (Other)
KW  - progressive supranuclear palsy (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:40884249
C2  - pmc:PMC12661634
DO  - DOI:10.1002/mds.70026
UR  - https://pub.dzne.de/record/282561
ER  -