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000271711 0247_ $$2doi$$a10.1002/mds.29866
000271711 0247_ $$2pmid$$apmid:38825840
000271711 0247_ $$2ISSN$$a0885-3185
000271711 0247_ $$2ISSN$$a1531-8257
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000271711 037__ $$aDZNE-2024-01063
000271711 041__ $$aEnglish
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000271711 1001_ $$0P:(DE-2719)9002627$$aQuattrone, Andrea$$b0$$udzne
000271711 245__ $$aMagnetic Resonance Imaging Measures to Track Atrophy Progression in Progressive Supranuclear Palsy in Clinical Trials.
000271711 260__ $$aNew York, NY$$bWiley$$c2024
000271711 3367_ $$2DRIVER$$aarticle
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000271711 3367_ $$00$$2EndNote$$aJournal Article
000271711 520__ $$aSeveral 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% atrophy progression, compared with other volumetric/planimetric measures and staging systems.This evidence may inform the selection of imaging endpoints to assess the treatment efficacy in reducing brain atrophy rate in PSP clinical trials, with automated atlas-based volumetry requiring smaller sample size than staging systems and planimetry to observe significant treatment effects. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
000271711 536__ $$0G:(DE-HGF)POF4-353$$a353 - Clinical and Health Care Research (POF4-353)$$cPOF4-353$$fPOF IV$$x0
000271711 588__ $$aDataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
000271711 650_7 $$2Other$$aatlas‐based volumetry
000271711 650_7 $$2Other$$aclinical trials
000271711 650_7 $$2Other$$aprogression
000271711 650_7 $$2Other$$aprogressive supranuclear palsy
000271711 650_7 $$2Other$$astaging system
000271711 650_2 $$2MeSH$$aAged
000271711 650_2 $$2MeSH$$aFemale
000271711 650_2 $$2MeSH$$aHumans
000271711 650_2 $$2MeSH$$aMale
000271711 650_2 $$2MeSH$$aMiddle Aged
000271711 650_2 $$2MeSH$$aAtrophy: pathology
000271711 650_2 $$2MeSH$$aBrain: diagnostic imaging
000271711 650_2 $$2MeSH$$aBrain: pathology
000271711 650_2 $$2MeSH$$aCohort Studies
000271711 650_2 $$2MeSH$$aDisease Progression
000271711 650_2 $$2MeSH$$aMagnetic Resonance Imaging: methods
000271711 650_2 $$2MeSH$$aSupranuclear Palsy, Progressive: diagnostic imaging
000271711 650_2 $$2MeSH$$aSupranuclear Palsy, Progressive: pathology
000271711 650_2 $$2MeSH$$aRandomized Controlled Trials as Topic
000271711 693__ $$0EXP:(DE-2719)DESCRIBE-PSP-20160101$$5EXP:(DE-2719)DESCRIBE-PSP-20160101$$eDZNE Clinical Registry Study of Neurodegenerative Diseases in Patients with Progressive Supranuclear Paresis (PSP)$$x0
000271711 7001_ $$aFranzmeier, Nicolai$$b1
000271711 7001_ $$aHuppertz, Hans-Jürgen$$b2
000271711 7001_ $$00000-0002-3054-9905$$aKlietz, Martin$$b3
000271711 7001_ $$aRoemer, Sebastian N$$b4
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000271711 7001_ $$0P:(DE-2719)2811373$$aHöglinger, Günter U$$b7$$eLast author$$udzne
000271711 7001_ $$aAL-108-231 Investigators, the Tauros MRI Investigators, the PASSPORT Study Group, the DESCRIBE-PSP Group$$b8$$eCollaboration Author
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