ORCID: https://orcid.org/0009-0004-4724-3824; Plan, Elodie L.
ORCID: https://orcid.org/0000-0002-2255-3904; Yousefi, Elham; König, Franz; Posch, Martin
ORCID: https://orcid.org/0000-0001-8499-8573; Hopfner, Franziska
ORCID: https://orcid.org/0000-0001-6524-0281; Höglinger, Günter
ORCID: https://orcid.org/0000-0001-7587-6187 und Karlsson, Mats O.
ORCID: https://orcid.org/0000-0003-1258-8297
(2024):
Quantitative Comparisons of Progressive Supranuclear Palsy Rating Scale Versions Using Item Response Theory.
In: Movement Disorders, Vol. 39, No. 12: pp. 2181-2189
[PDF, 1MB]

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.
Item Type: | Journal article |
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Faculties: | Medicine > Munich Cluster for Systems Neurology (SyNergy) Medicine > Institute for Stroke and Dementia Research (ISD) Medicine > Medical Center of the University of Munich > Neurological Clinic and Polyclinic with Friedrich Baur Institute |
Subjects: | 600 Technology > 610 Medicine and health |
URN: | urn:nbn:de:bvb:19-epub-123130-0 |
ISSN: | 0885-3185 |
Language: | English |
Item ID: | 123130 |
Date Deposited: | 13. Dec 2024 15:16 |
Last Modified: | 07. Jan 2025 15:28 |
DFG: | Gefördert durch die Deutsche Forschungsgemeinschaft (DFG) - 390857198 |