TY  - JOUR
AU  - Quattrone, Andrea
AU  - Franzmeier, Nicolai
AU  - Huppertz, Hans-Jürgen
AU  - Klietz, Martin
AU  - Roemer, Sebastian N
AU  - Boxer, Adam L
AU  - Levin, Johannes
AU  - Höglinger, Günter U
TI  - Magnetic Resonance Imaging Measures to Track Atrophy Progression in Progressive Supranuclear Palsy in Clinical Trials.
JO  - Movement disorders
VL  - 39
IS  - 8
SN  - 0885-3185
CY  - New York, NY
PB  - Wiley
M1  - DZNE-2024-01063
SP  - 1329 - 1342
PY  - 2024
AB  - Several magnetic resonance imaging (MRI) measures have been suggested as progression biomarkers in progressive supranuclear palsy (PSP), and some PSP staging systems have been recently proposed.Comparing structural MRI measures and staging systems in tracking atrophy progression in PSP and estimating the sample size to use them as endpoints in clinical trials.Progressive supranuclear palsy-Richardson's syndrome (PSP-RS) patients with one-year-follow-up longitudinal brain MRI were selected from the placebo arms of international trials (NCT03068468, NCT01110720, NCT01049399) and the DescribePSP cohort. The discovery cohort included patients from the NCT03068468 trial; the validation cohort included patients from other sources. Multisite age-matched healthy controls (HC) were included for comparison. Several MRI measures were compared: automated atlas-based volumetry (44 regions), automated planimetric measures of brainstem regions, and four previously described staging systems, applied to volumetric data.Of 508 participants, 226 PSP patients including discovery (n = 121) and validation (n = 105) cohorts, and 251 HC were included. In PSP patients, the annualized percentage change of brainstem and midbrain volume, and a combined index including midbrain, frontal lobe, and third ventricle volume change, were the progression biomarkers with the highest effect size in both cohorts (discovery: >1.6; validation cohort: >1.3). These measures required the lowest sample sizes (n < 100) to detect 30
KW  - Aged
KW  - Female
KW  - Humans
KW  - Male
KW  - Middle Aged
KW  - Atrophy: pathology
KW  - Brain: diagnostic imaging
KW  - Brain: pathology
KW  - Cohort Studies
KW  - Disease Progression
KW  - Magnetic Resonance Imaging: methods
KW  - Supranuclear Palsy, Progressive: diagnostic imaging
KW  - Supranuclear Palsy, Progressive: pathology
KW  - Randomized Controlled Trials as Topic
KW  - atlas‐based volumetry (Other)
KW  - clinical trials (Other)
KW  - progression (Other)
KW  - progressive supranuclear palsy (Other)
KW  - staging system (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:38825840
DO  - DOI:10.1002/mds.29866
UR  - https://pub.dzne.de/record/271711
ER  -