Clinical correlates of longitudinal brain atrophy in progressive supranuclear palsy

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 internatio...

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Bibliographic Details
Published in:Parkinsonism & related disorders 2016-07, Vol.28, p.29-35
Main Authors: Tsai, Richard M, Lobach, Iryna, Bang, Jee, Whitwell, Jennifer L, Senjem, Matthew L, Jack, Clifford R, Rosen, Howard, Miller, Bruce, Boxer, Adam L
Format: Article
Language:English
Subjects:
Atrophy - pathology
Biomarkers
Brain - diagnostic imaging
Brain - pathology
Clinical trials
Disease Progression
Female
Humans
Imaging
Magnetic Resonance Imaging - methods
Male
Middle Aged
MRI
Neurology
Progressive supranuclear palsy
Supranuclear Palsy, Progressive - diagnostic imaging
Supranuclear Palsy, Progressive - pathology
Supranuclear Palsy, Progressive - physiopathology
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Summary: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 
ISSN:1353-8020
1873-5126
DOI:10.1016/j.parkreldis.2016.04.006