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Piot, Ines; Schweyer, Kerstin; Respondek, Gesine; Stamelou, Maria; Gasser, Thomas; Hermann, Andreas; Höglinger, Gunter; Hoellerhage, Matthias; Kimmich, Okka; Klockgether, Thomas; Levin, Johannes; Machetanz, Gerrit; Osterrath, Antje; Palleis, Carla; Prudlo, Johannes; Spottke, Annika; Berg, Daniela; Buerk, Katrin; Classen, Joseph; Eggers, Carsten; Greuel, Andrea; Grimm, Max-Joseph; Hermann, Lennard; Iankova, Vassilena; Jahn, Klaus; Jost, Wolfgang; Klietz, Martin; Kuehn, Andrea; Marxreiter, Franz; Paschen, Steffen; Poetter-Nerger, Monika; Preisl, Marie-Therese; Prilop, Lisa; Toenges, Lars; Trenkwalder, Claudia; Warnecke, Tobias; Wegner, Florian; Winkler, Jürgen; Antonini, Angelo; Bhatia, Kailash P.; Boxer, Adam L.; Colosimo, Carlo; Compta, Yaroslau; Corvol, Jean-Christophe; Golbe, Lawrence I.; Hoglinger, Guenter U.; Lang, Anthony E.; Litvan, Irene; Morris, Huw R.; Nilsson, Christer; Pantelyat, Alexander; Respondek, Gesine; Stamelou, Maria; Sckopke, Philipp; Schenk, Thomas; Goetz, Christopher G. und Stebbins, Glenn T. (2020): The Progressive Supranuclear Palsy Clinical Deficits Scale. In: Movement Disorders, Bd. 35, Nr. 4: S. 650-661

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Abstract

Background: There is currently no undisputed, validated, clinically meaningful measure for deficits in the broad spectrum of PSP phenotypes. Objective: To develop a scale to monitor clinical deficits in patients with PSP across its broad phenotypes. Methods The Progressive Supranuclear Palsy Clinical Deficits Scale was conceptualized to cover seven clinical domains (Akinesia-rigidity, Bradyphrenia, Communication, Dysphagia, Eye movements, Finger dexterity, and Gait & balance), each scored from 0 to 3 (no, mild, moderate, or severe deficits). User guidelines were developed to standardize its application. Progressive Supranuclear Palsy Clinical Deficits Scale scores were collected in patients fulfilling the MDS-PSP diagnostic criteria in two independent, multicenter, observational studies, both cross-sectionally (exploratory DescribePSP cohort;confirmatory ProPSP cohort) and longitudinally (12-months' follow-up, both cohorts). Results Cognitive pretesting demonstrated easy scale utility. In total, 164 patients were scored (70.4 +/- 7.6 years;62% males, 35% variant phenotypes). Mean Progressive Supranuclear Palsy Clinical Deficits Scale completion time was 4 minutes. The Progressive Supranuclear Palsy Clinical Deficits Scale total score correlated with existing scales (e.g., Progressive Supranuclear Palsy Rating Scale: R = 0.88;P < 0.001). Individual Progressive Supranuclear Palsy Clinical Deficits Scale items correlated well with similar constructs in existing scales. Internal consistency (Cronbach's alpha: 0.75), inter-rater reliability (0.96), and test-retest stability (0.99) were acceptable. The PSP-CDS showed significant 12-month change (baseline, 8.6 +/- 3.6;follow-up: 10.8 +/- 3.6;annualized difference: 3.4 +/- 3.4;n = 49;P < 0.0001). Sample sizes required per arm for a two-arm, 1-year follow-up therapeutic trial to detect 50% change in Progressive Supranuclear Palsy Clinical Deficits Scale progression was estimated to be 65 (two-sided, two-sample t test). Conclusion The Progressive Supranuclear Palsy Clinical Deficits Scale is a rapidly completed, clinimetrically sound scale for clinical care and research involving PSP. (c) 2020 International Parkinson and Movement Disorder Society

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