Quantitative Comparisons of Progressive Supranuclear Palsy Rating Scale Versions Using Item Response Theory

Gewily, Mohamed ; Plan, Elodie L. ; Yousefi, Elham; König, Franz; Posch, Martin ; Hopfner, Franziska ; Höglinger, Günter und Karlsson, Mats O. (2024): Quantitative Comparisons of Progressive Supranuclear Palsy Rating Scale Versions Using Item Response Theory. In: Movement Disorders [Forthcoming]

Abstract

Background : Progressive supranuclear palsy (PSP) is a neurodegenerative, late-onset disease that is challenging in terms of assessment. The Progressive Supranuclear Palsy Rating Scale (PSPRS), a 28-item clinician-reported scale, is the most established clinical outcome assessment method. Recently, the U.S. Food and Drug Administration (FDA) has proposed a subscale of 10 items as an alternative to full PSPRS. Objectives : To quantitatively evaluate and compare the properties of full PSPRS and the FDA subscale using item response theory. To develop a progression model of the disease and assess relative merits of study designs and analysis options. Methods : Data of 979 patients from four interventional trials and two registries were available for analysis. Our investigation was divided into: (1) estimating informativeness of the 28 items; (2) estimating disease progression; and (3) comparing the scales, trial designs, and analysis options with respect to power to detect a clinically relevant treatment effect. Results : PSPRS item scores had a low pairwise correlation (r = 0.17 ± 0.14) and the items irritability, sleep difficulty, and postural tremor were uncorrelated with the other items. The FDA-selected items displayed higher correlation (r = 0.35 ± 0.14) and were the basis for a longitudinal item response model including disease progression. Trial simulations indicated that identification of a disease-modifying treatment effect required less than half the study size if the analysis was based on longitudinal item information compared with total scores at end-of-treatment. Conclusion : A longitudinal item response model based on the FDA-selected PSPRS items is a promising tool in evaluating treatments for PSP.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin > Munich Cluster for Systems Neurology (SyNergy) ; Medizin > Institut für Schlaganfall- und Demenzforschung (ISD) ; Medizin > Klinikum der LMU München > Neurologische Klinik und Poliklinik mit Friedrich-Baur-Institut
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0885-3185
• Sprache: Englisch
• DFG: Gefördert durch die Deutsche Forschungsgemeinschaft (DFG) - 390857198