Magnetic Resonance Imaging Measures to Track Atrophy Progression in Progressive Supranuclear Palsy in Clinical Trials
dc.rights.license | open | en_US |
dc.contributor.author | QUATTRONE, Andrea | |
dc.contributor.author | FRANZMEIER, Nicolai | |
dc.contributor.author | HUPPERTZ, Hans-Jurgen | |
dc.contributor.author | KLIETZ, Martin | |
dc.contributor.author | ROEMER, Sebastian N | |
dc.contributor.author | BOXER, Adam L | |
dc.contributor.author | LEVIN, Johannes | |
dc.contributor.author | HOGLINGER, Gunter U | |
hal.structure.identifier | Bordeaux population health [BPH] | |
dc.contributor.author | SAMIER FOUBERT, Alexandra | |
dc.date.accessioned | 2025-02-20T15:23:21Z | |
dc.date.available | 2025-02-20T15:23:21Z | |
dc.date.issued | 2024-08-01 | |
dc.identifier.issn | 1531-8257 | en_US |
dc.identifier.uri | https://oskar-bordeaux.fr/handle/20.500.12278/205067 | |
dc.description.abstractEn | Background: 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. Objective: 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. Methods: 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. Results: 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. Conclusions: 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. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society. | |
dc.language.iso | EN | en_US |
dc.rights | Attribution-NonCommercial-NoDerivs 3.0 United States | * |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-nd/3.0/us/ | * |
dc.subject.en | Atlas‐Based Volumetry | |
dc.subject.en | Clinical Trials | |
dc.subject.en | Progression | |
dc.subject.en | Progressive Supranuclear Palsy | |
dc.subject.en | Staging System | |
dc.title.en | Magnetic Resonance Imaging Measures to Track Atrophy Progression in Progressive Supranuclear Palsy in Clinical Trials | |
dc.title.alternative | Mov Disord | en_US |
dc.type | Article de revue | en_US |
dc.identifier.doi | 10.1002/mds.29866 | en_US |
dc.subject.hal | Sciences du Vivant [q-bio]/Santé publique et épidémiologie | en_US |
dc.identifier.pubmed | 38825840 | en_US |
bordeaux.journal | Movement Disorders | en_US |
bordeaux.page | 1329-1342 | en_US |
bordeaux.volume | 39 | en_US |
bordeaux.hal.laboratories | Bordeaux Population Health Research Center (BPH) - UMR 1219 | en_US |
bordeaux.issue | 8 | en_US |
bordeaux.institution | Université de Bordeaux | en_US |
bordeaux.institution | INSERM | en_US |
bordeaux.team | Coll_ACTIVE_BPH | en_US |
bordeaux.peerReviewed | oui | en_US |
bordeaux.inpress | non | en_US |
hal.identifier | hal-04959334 | |
hal.version | 1 | |
hal.date.transferred | 2025-02-20T15:23:24Z | |
hal.popular | non | en_US |
hal.audience | Internationale | en_US |
hal.export | true | |
dc.rights.cc | Pas de Licence CC | en_US |
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