1. Progression of two Progressive Supranuclear Palsy phenotypes with comparable initial disability
- Author
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Shoeibi, A, Litvan, I, Tolosa, E, del Ser, T, Lee, E, Gomez, JC, Tijero, B, Berganzo, K, de Yebenes, JG, Sendon, JLL, Garcia, G, Buongiorno, MT, Bargallo, N, Burguera, JA, Martinez, I, Ruiz-Martinez, J, Narrativel, I, Vivancos, F, Ybot, I, Aguilar, M, Quilez, P, Boada, M, Lafuente, A, Hernandez, I, Lopez-Lozano, JJ, Mata, M, Kupsch, A, Lipp, A, Ebersbach, G, Schmidt, T, Hahn, K, Heoglinger, G, Hollerhage, M, Oertel, WH, Respondek, G, Stamelou, M, Reichmann, H, Wolz, M, Schneider, C, Klingelhofer, L, Berg, D, Maetzler, W, Srulijes, KK, Ludolph, A, Kassubek, J, Steiger, M, Tyler, K, Burn, DJ, Morris, L, Lees, A, Ling, H, Hauser, R, McClain, T, Truong, D, Jenkins, S, Houghton, D, Ferrara, J, Bordelon, Y, Gratiano, A, Golbe, L, Mark, M, Uitti, R, Gerpen, JV, and TAUROS Investigators
- Subjects
Progression ,Progressive supranuclear palsy ,Progressive supranuclear palsy-Richardson syndrome ,Progressive supranuclear palsy-Parkinsonism ,eye diseases - Abstract
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.
- Published
- 2019