Journal
PARKINSONISM & RELATED DISORDERS
Volume 66, Issue -, Pages 87-93Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.parkreldis.2019.07.010
Keywords
Progression; Progressive supranuclear palsy; Progressive supranuclear palsy-Parkinsonism; Progressive supranuclear palsy-Richardson syndrome
Categories
Funding
- Noscira SA
- Parkinson 1 Study Group
- Michael J. Fox Foundation
- AVID Pharmaceuticals
- Abbvie/C2N Diagnostics
- Biogen/Bristol Myers Squibb
- Spanish Network for Research on Neurodegenerative Disorders (CIBERNED) Instituto Carlos III (ISCIII)
- Michael J. Fox Foundation for Parkinson's Research (MJFF)
- Fondo de Investigacioncs Sanitarias de la Seguridad Social (FISS)
- [5P50 AG005131.31]
- [5T35IIL007491]
- [1U01NS086659]
- [1U54NS092089-01]
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Introduction: To avoid bias and optimize statistical power of disease-modifying therapeutic trials, it is critical to include homogeneous populations with similar rate of progression over time. Patients with Progressive Supranuclear Palsy (PSP)-Parkinsonism phenotype have overall slower disease progression than those with PSP-Richardson syndrome phenotype. However, it is unclear if the progression rate of PSP Parkinsonism is the same when the PSP-Parkinsonism converts to PSP Richardson syndrome. We aimed to determine and compare disease progression rate of patients with the two most common PSP phenotypes: PSP-Parkinsonism and PSP Richardson syndrome, participating in the TAUROS trial. Methods: 138 patients, 56 with PSP-Parkinsonism and 82 with PSP-Richardson syndrome, with similar clinical severity at baseline, were followed up to 60 weeks. PSP-Parkinsonism allocation was based on experts' judgement and PSP-Richardson on probable NINDS-PSP criteria. Global disease progression was measured by the PSP Rating Scale as primary outcome measure and several secondary outcome measures. Results: PSP-Richardson syndrome patients had significantly faster progression based on the primary and three secondary outcome measures: the Dementia Rating Scale-2, Frontal Assessment Battery, and lexical fluency scale. Analyses including only patients with a baseline symptom duration under five years showed similar results. PSP phenotype was the strongest predictor for disease progression. Conclusion: This research showed that even when disease severity and clinical features at baseline arc similar, patients with PSP- Richardson syndrome progressed significantly faster than those with PSP-Parkinsonism. Therefore, unless stratified by phenotype, future therapeutic clinical trials should not lump PSP patients with these phenotypes as a single disorder even if they have similar disease severity at screening.
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