{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T15:17:05Z","timestamp":1788967025171,"version":"build-2803163510"},"reference-count":25,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T00:00:00Z","timestamp":1741046400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100008909","name":"Once Upon A Time Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008909","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002243","name":"National Ataxia Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002243","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":[[2025,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The Patient\u2010Reported Outcome Measure of Ataxia (PROM\u2010Ataxia) has been validated cross\u2010sectionally but not longitudinally.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>We aimed to validate PROM\u2010Ataxia as a measure of patient experience of disease over time, examine overall and domain\u2010specific progression, and test convergent validity with other clinical outcome assessments (COAs).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We derived PROM\u2010Ataxia data from 176 patients with spinocerebellar ataxia types 1, 2, 3, 6, 7, 8, or 10 in the Clinical Research Consortium for the Study of Cerebellar Ataxia at baseline and 1 year. We classified patients' ataxia severity stage (\u201cseverity\u201d) according to the Friedreich's Ataxia Rating Scale Functional Staging into <jats:italic>mild<\/jats:italic>, <jats:italic>moderate<\/jats:italic>, and <jats:italic>severe<\/jats:italic> subgroups. Analyses of the entire cohort and by severity subgroup included internal consistency, sensitivity to disease severity, predictive modeling of score changes, correlations with COAs: Brief Ataxia Rating Scale, Scale for Assessment and Rating of Ataxia, Fatigue Severity Scale, Cerebellar Cognitive Affective Syndrome scale, EuroQol 5\u2010Dimension, and responsiveness to disease progression.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The PROM\u2010Ataxia exhibited high internal consistency and correlated with other COAs. Scores demonstrated sensitivity to disease severity and evolving patient experience. Progression was sigmoidal, with the greatest change in <jats:italic>moderate<\/jats:italic> patients. Compared with other COAs, PROM\u2010Ataxia captured the most change. Mental features worsened fastest in <jats:italic>mild<\/jats:italic> patients, physical in <jats:italic>moderate<\/jats:italic> patients, and activities of daily living in <jats:italic>severe<\/jats:italic> patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>PROM\u2010Ataxia is more sensitive to change than ataxia COAs, captures the evolution of patients' experience of disease over 1\u2009year, and reveals domain\u2010specific progression. Studies of larger cohorts and different ataxia diagnoses over longer periods may provide insights to further enhance clinical care and research. \u00a9 2025 International Parkinson and Movement Disorder Society.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mds.30158","type":"journal-article","created":{"date-parts":[[2025,3,11]],"date-time":"2025-03-11T17:38:58Z","timestamp":1741714738000},"page":"917-927","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Spinocerebellar Ataxia Progression Measured with the Patient\u2010Reported Outcome Measure of Ataxia"],"prefix":"10.1002","volume":"40","author":[{"given":"Anna L.","family":"Burt","sequence":"first","affiliation":[{"name":"Ataxia Center, Department of Neurology Massachusetts General Hospital and Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gilbert","family":"L'Italien","sequence":"additional","affiliation":[{"name":"Biohaven Pharmaceuticals  New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan L.","family":"Perlman","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5140-3967","authenticated-orcid":false,"given":"Liana S.","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Department of Neurology Johns Hopkins School of Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheng\u2010Han","family":"Kuo","sequence":"additional","affiliation":[{"name":"Department of Neurology Columbia University Medical Center  New York City New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7180-2869","authenticated-orcid":false,"given":"Tetsuo","family":"Ashizawa","sequence":"additional","affiliation":[{"name":"Stanley H. Appel Department of Neurology Houston Methodist Hospital  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0640-1641","authenticated-orcid":false,"given":"Theresa","family":"Zesiewicz","sequence":"additional","affiliation":[{"name":"Department of Neurology University of South Florida  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cameron","family":"Dietiker","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Puneet","family":"Opal","sequence":"additional","affiliation":[{"name":"Department of Neurology Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antoine","family":"Duquette","sequence":"additional","affiliation":[{"name":"Service de Neurologie, Centre Hospitalier de l'Universit\u00e9 de Montr\u00e9al (CHUM) University of Montreal  Montreal Quebec Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"George R.","family":"Wilmot","sequence":"additional","affiliation":[{"name":"Department of Neurology Emory University  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vikram G.","family":"Shakkottai","sequence":"additional","affiliation":[{"name":"Department of Neurology and O'Donnell Brain Institute UT Southwestern Medical Center  Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher M.","family":"Gomez","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Chicago  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharan R.","family":"Srinivasan","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Michigan  Ann Arbor Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry","family":"Paulson","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Michigan  Ann Arbor Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael D.","family":"Geschwind","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandie","family":"Worley","sequence":"additional","affiliation":[{"name":"Department of Neurology Columbia University Medical Center  New York City New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiadi U.","family":"Onyike","sequence":"additional","affiliation":[{"name":"Department of Neurology Johns Hopkins School of Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Billnitzer","sequence":"additional","affiliation":[{"name":"Stanley H. 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