{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T14:19:05Z","timestamp":1785161945483,"version":"3.55.0"},"reference-count":28,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2024,8,29]],"date-time":"2024-08-29T00:00:00Z","timestamp":1724889600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"publisher","award":["EXC 2145 SyNergy \u2010 ID 390857198"],"award-info":[{"award-number":["EXC 2145 SyNergy \u2010 ID 390857198"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004359","name":"Vetenskapsr\u00e5det","doi-asserted-by":"publisher","award":["2018\u201003317"],"award-info":[{"award-number":["2018\u201003317"]}],"id":[{"id":"10.13039\/501100004359","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["movementdisorders.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Movement Disorders"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Progressive supranuclear palsy (PSP) is a neurodegenerative, late\u2010onset disease that is challenging in terms of assessment. The Progressive Supranuclear Palsy Rating Scale (PSPRS), a 28\u2010item clinician\u2010reported 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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>PSPRS item scores had a low pairwise correlation (r\u2009=\u20090.17\u2009\u00b1\u20090.14) and the items irritability, sleep difficulty, and postural tremor were uncorrelated with the other items. The FDA\u2010selected items displayed higher correlation (r\u2009=\u20090.35\u2009\u00b1\u20090.14) and were the basis for a longitudinal item response model including disease progression. Trial simulations indicated that identification of a disease\u2010modifying treatment effect required less than half the study size if the analysis was based on longitudinal item information compared with total scores at end\u2010of\u2010treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>A longitudinal item response model based on the FDA\u2010selected PSPRS items is a promising tool in evaluating treatments for PSP. \u00a9 2024 The Author(s). <jats:italic>Movement Disorders<\/jats:italic> published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mds.30001","type":"journal-article","created":{"date-parts":[[2024,8,29]],"date-time":"2024-08-29T07:54:38Z","timestamp":1724918078000},"page":"2181-2189","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Quantitative Comparisons of Progressive Supranuclear Palsy Rating Scale Versions Using Item Response Theory"],"prefix":"10.1002","volume":"39","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-4724-3824","authenticated-orcid":false,"given":"Mohamed","family":"Gewily","sequence":"first","affiliation":[{"name":"Department of Pharmacy Uppsala University  Uppsala Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2255-3904","authenticated-orcid":false,"given":"Elodie L.","family":"Plan","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Uppsala University  Uppsala Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elham","family":"Yousefi","sequence":"additional","affiliation":[{"name":"Center for Medical Data Science Medical University of Vienna  Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franz","family":"K\u00f6nig","sequence":"additional","affiliation":[{"name":"Center for Medical Data Science Medical 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Ludwig\u2010Maximilians\u2010Universit\u00e4t (LMU) M\u00fcnchen  Munich Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  Munich Germany"},{"name":"Munich Cluster for Systems Neurology (SyNergy)  Munich Germany"},{"name":"Department of Neurology Hannover Medical School  Hannover Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1258-8297","authenticated-orcid":false,"given":"Mats O.","family":"Karlsson","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Uppsala University  Uppsala 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[cited Jul 18 2024]; Available from:https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/submitting-clinical-trial-datasets-and-documentation-clinical-outcome-assessments-using-item."},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1208\/s12248-017-0058-8"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11095-014-1315-5"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1208\/s12248-016-9977-z"},{"key":"e_1_2_9_29_1","unstructured":"YousefiE GewilyM K\u00f6nigF H\u00f6glingerG HopfnerF KarlssonMO et al.Efficiency of multivariate tests in trials in progressive supranuclear palsy [Internet].arXiv 2023. 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