{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,23]],"date-time":"2026-01-23T19:18:08Z","timestamp":1769195888543,"version":"3.49.0"},"reference-count":34,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2021,7,15]],"date-time":"2021-07-15T00:00:00Z","timestamp":1626307200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurol."],"abstract":"<jats:p><jats:bold>Introduction:<\/jats:bold> An understanding of the clinimetric properties of clinical assessments, including their constraints, is critical to sound clinical study and trial design. Utilizing data from Enroll-HD\u2014a global, prospective HD observational study and clinical research platform\u2014we examined several well-established HD clinical assessments across all stages of disease for evidence of instrument constraints, specifically floor\/ceiling effects, to inform selection of appropriate instruments for use in future studies\/trials and identify gaps in instrument utility over the life-course of the disease.<\/jats:p><jats:p><jats:bold>Material and Methods:<\/jats:bold> Analyzing publicly available data from 6,614 HD gene-expansion carriers (HDGECs), we grouped participants into deciles based on baseline CAP score, which ranged from 26 to 229. We used descriptive statistics to characterize data distribution for 25 outcome measures (encompassing motor, function, cognition, and psychiatric\/behavioral domains) in each CAP decile. A skewness statistic threshold of \u00b12 was defined <jats:italic>a priori<\/jats:italic> to indicate floor\/ceiling effects.<\/jats:p><jats:p><jats:bold>Results:<\/jats:bold> We found evidence of floor\/ceiling effects in the early premanifest stages of disease for most motor and function assessments (e.g., TMS, TFC) and select cognitive tasks (MMSE, Trail Making tests). Other cognitive assessments, and the HADS-SIS scales, performed well ubiquitously, with no evidence of floor\/ceiling effects at any disease stage. Floor\/ceiling effects were evident at every disease stage for certain assessments, including PBA-s measures. Ceiling effects were apparent for DCL from onset stages onwards, as expected.<\/jats:p><jats:p><jats:bold>Discussion:<\/jats:bold> Developing instruments sensitive to subtle differences in performance at the earlier stages of the disease spectrum, particularly in motor and function domains, is warranted.<\/jats:p>","DOI":"10.3389\/fneur.2021.595679","type":"journal-article","created":{"date-parts":[[2021,7,15]],"date-time":"2021-07-15T18:10:48Z","timestamp":1626372648000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":12,"title":["Utility of Huntington's Disease Assessments by Disease Stage: Floor\/Ceiling Effects"],"prefix":"10.3389","volume":"12","author":[{"given":"Daisy","family":"Abreu","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Ware","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nellie","family":"Georgiou-Karistianis","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Blair R.","family":"Leavitt","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cheryl J.","family":"Fitzer-Attas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raquel","family":"Lobo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ana Raquel","family":"Fernandes","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olivia","family":"Handley","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karen E.","family":"Anderson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie C.","family":"Stout","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina","family":"Sampaio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2021,7,15]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"218","DOI":"10.1016\/S0140-6736(07)60111-1","article-title":"Huntington's disease","volume":"369","author":"Walker","year":"2007","journal-title":"Lancet."},{"key":"B2","doi-asserted-by":"publisher","first-page":"874","DOI":"10.1136\/jnnp.2007.128728","article-title":"Detection of Huntington's disease decades before diagnosis: the Predict-HD study","volume":"79","author":"Paulsen","year":"2008","journal-title":"J Neurol Neurosurg Psychiatry."},{"key":"B3","doi-asserted-by":"publisher","first-page":"737","DOI":"10.1002\/mdc3.12502","article-title":"Survival, mortality, causes and places of death in a European Huntington's disease prospective cohort","volume":"4","author":"Rodrigues","year":"2017","journal-title":"Mov Disord Clin Pract."},{"key":"B4","article-title":"APA Dictionary of Statistics and Research Methods","author":"Guha","year":"2014"},{"key":"B5","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1002\/mdc3.12388","article-title":"Data analytics from Enroll-HD, a global clinical research platform for Huntington's disease","volume":"4","author":"Landwehrmeyer","year":"2017","journal-title":"Mov Disord Clin Pract."},{"key":"B6","unstructured":"2015"},{"key":"B7","article-title":"UHDRS Total Motor Score 0-124","year":"1999"},{"key":"B8","article-title":"UHDRS Diagnostic Confidence Level 0-4","year":"1999"},{"key":"B9","article-title":"UHDRS Total Functional Capacity 0-13","year":"1999"},{"key":"B10","article-title":"UHDRS Functional Assessment Scale 0-25","year":"1999"},{"key":"B11","article-title":"UHDRS Independence Scale 5-100","year":"1999"},{"key":"B12","doi-asserted-by":"publisher","first-page":"142","DOI":"10.1111\/j.1532-5415.1991.tb01616.x","article-title":"The timed \u201cUp & Go\u201d: a test of basic functional mobility for frail elderly persons","volume":"39","author":"Podsiadlo","year":"1991","journal-title":"J. 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