eprintid: 10055847 rev_number: 26 eprint_status: archive userid: 608 dir: disk0/10/05/58/47 datestamp: 2018-09-13 15:41:48 lastmod: 2021-10-15 22:32:11 status_changed: 2018-09-13 15:41:48 type: article metadata_visibility: show creators_name: Tsai, RM creators_name: Lobach, I creators_name: Bang, J creators_name: Whitwell, JL creators_name: Senjem, ML creators_name: Jack, CR creators_name: Rosen, H creators_name: Miller, B creators_name: Boxer, AL creators_name: AL-108-231 Investigators, title: Clinical correlates of longitudinal brain atrophy in progressive supranuclear palsy ispublished: pub divisions: UCL divisions: B02 divisions: C07 divisions: D07 divisions: F84 keywords: Biomarkers, Clinical trials, Imaging, MRI, Progressive supranuclear palsy, Atrophy, Biomarkers, Brain, Disease Progression, Female, Humans, Magnetic Resonance Imaging, Male, Middle Aged, Supranuclear Palsy, Progressive note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions abstract: INTRODUCTION: There are no effective treatments for progressive supranuclear palsy (PSP). Volumetric MRI (vMRI) may be a useful surrogate outcome measure in PSP clinical trials. The goal of the study was to evaluate the potential of vMRI to correlate with clinical outcomes from an international clinical trial population. METHODS: PSP patients (n = 198) from the AL-108-231 trial who had high quality vMRI and Progressive Supranuclear Palsy Rating Scale (PSPRS), Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), Schwab and England Activities of Daily Living (SEADL), Color Trails Test, Geriatric Depression Screen (GDS) and one year Clinician Global Impression of Change (CGIC) data from the baseline and 52 week visits were included. Linear regression was used to relate baseline values and annual clinical rating scale changes to annual regional vMRI changes (whole brain, ventricular, midbrain and superior cerebellar peduncle volumes). RESULTS: Effect sizes (Cohen's d) measuring disease progression over one year were largest for vMRI (midbrain [1.27] and ventricular volume [1.31]) but similar to PSPRS (1.26). After multiple comparison adjustment, annual changes in PSPRS, RBANS, SEADL, Color Trails Test, GDS and one year CGIC were modestly correlated with annual vMRI changes (p < 0.05). Baseline neuropsychological status on RBANS (p = 0.019) and Color Trails (p < 0.01) predicted annual midbrain atrophy rates. CONCLUSION: Standard vMRI measurements are sensitive to disease progression in large, multicenter PSP clinical trials, but are not well correlated with clinical changes. vMRI changes may be useful as supportive endpoints in PSP trials. date: 2016-04-24 date_type: published official_url: https://doi.org/10.1016/j.parkreldis.2016.04.006 oa_status: green full_text_type: other pmcid: PMC4914401 language: eng primo: open primo_central: open_green article_type_text: Clinical Trial, Phase II verified: verified_manual elements_id: 1574637 doi: 10.1016/j.parkreldis.2016.04.006 pii: S1353-8020(16)30084-0 lyricists_name: Lees, Andrew lyricists_id: AJLEE60 actors_name: Bracey, Alan actors_id: ABBRA90 actors_role: owner full_text_status: public publication: Parkinsonism & Related Disorders volume: 28 pagerange: 29-35 event_location: England issn: 1873-5126 citation: Tsai, RM; Lobach, I; Bang, J; Whitwell, JL; Senjem, ML; Jack, CR; Rosen, H; ... AL-108-231 Investigators; + view all <#> Tsai, RM; Lobach, I; Bang, J; Whitwell, JL; Senjem, ML; Jack, CR; Rosen, H; Miller, B; Boxer, AL; AL-108-231 Investigators; - view fewer <#> (2016) Clinical correlates of longitudinal brain atrophy in progressive supranuclear palsy. Parkinsonism & Related Disorders , 28 pp. 29-35. 10.1016/j.parkreldis.2016.04.006 <https://doi.org/10.1016/j.parkreldis.2016.04.006>. Green open access document_url: https://discovery.ucl.ac.uk/id/eprint/10055847/1/nihms-785506.pdf