{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,7]],"date-time":"2026-11-07T18:21:51Z","timestamp":1794075711773,"version":"4.8.0"},"reference-count":19,"publisher":"SAGE Publications","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JHD"],"published-print":{"date-parts":[[2022,6,3]]},"abstract":"<jats:p>Background: When developed properly, disease-specific patient reported outcome measures have the potential to measure relevant changes in how a patient feels and functions in the context of a therapeutic trial. The Huntington\u2019s Disease Health Index (HD-HI) is a multifaceted disease-specific patient reported outcome measure (PROM) designed specifically to satisfy previously published FDA guidance for developing PROMs for product development and labeling claims. Objective: In preparation for clinical trials, we examine the validity, reliability, clinical relevance, and patient understanding of the Huntington\u2019s Disease Health Index (HD-HI). Methods: We partnered with 389 people with Huntington\u2019s disease (HD) and caregivers to identify the most relevant questions for the HD-HI. We subsequently utilized two rounds of factor analysis, cognitive interviews with fifteen individuals with HD, and test-retest reliability assessments with 25 individuals with HD to refine, evaluate, and optimize the HD-HI. Lastly, we determined the capability of the HD-HI to differentiate between groups of HD participants with high versus low total functional capacity score, prodromal versus manifest HD, and normal ambulation versus mobility impairment. Results: HD participants identified 13 relevant and unique symptomatic domains to be included as subscales in the HD-HI. All HD-HI subscales had a high level of internal consistency and reliability and were found by participants to have acceptable content, relevance, and usability. The total HD-HI score and each subscale score statistically differentiated between groups of HD participants with high versus low disease burden. Conclusion: Initial evaluation of the HD-HI supports its validity and reliability as a PROM for assessing how individuals with HD feel and function.<\/jats:p>","DOI":"10.3233\/jhd-210506","type":"journal-article","created":{"date-parts":[[2022,5,3]],"date-time":"2022-05-03T11:46:00Z","timestamp":1651578360000},"page":"217-226","source":"Crossref","is-referenced-by-count":11,"title":["The Huntington\u2019s Disease Health Index: Initial Evaluation of a Disease-Specific Patient Reported Outcome Measure"],"prefix":"10.1177","volume":"11","author":[{"given":"Olivia S.","family":"Brumfield","sequence":"first","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine E.","family":"Zizzi","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"},{"name":"School of Public and International Affairs, Princeton University, Princeton, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nuran","family":"Dilek","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danae G.","family":"Alexandrou","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alistair M.","family":"Glidden","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Spencer","family":"Rosero","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Weinstein","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jamison","family":"Seabury","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron","family":"Kaat","sequence":"additional","affiliation":[{"name":"Department of Medical Sciences, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael P.","family":"McDermott","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"},{"name":"Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E. Ray","family":"Dorsey","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"},{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chad R.","family":"Heatwole","sequence":"additional","affiliation":[{"name":"Center for Health + Technology, University of Rochester, Rochester, NY, USA"},{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"key":"10.3233\/JHD-210506_ref1","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1136\/jmg.31.11.872","article-title":"Trinucleotide repeat length and progression of illness in Huntington\u2019s disease","volume":"31","author":"Kieburtz","year":"1994","journal-title":"J Med 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Nerve"},{"issue":"2","key":"10.3233\/JHD-210506_ref13","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1002\/ana.25282","article-title":"The Charcot-Marie-Tooth Health Index: Evaluation of a patient-reported outcome","volume":"84","author":"Johnson","year":"2018","journal-title":"Ann Neurol"},{"issue":"6","key":"10.3233\/JHD-210506_ref14","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1002\/mus.27223","article-title":"The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions","volume":"63","author":"Zizzi","year":"2021","journal-title":"Muscle Nerve"},{"issue":"1","key":"10.3233\/JHD-210506_ref15","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1002\/mus.26066","article-title":"Myotonic dystrophy patient preferences in patient-reported outcome measures","volume":"58","author":"Heatwole","year":"2018","journal-title":"Muscle 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Guidance for industry patient-reported outcome measures: Use in medical product development to support labeling claims. Silver Spring, MD: Center for Drug Evaluation and Research. Center for Biologics Evaluation and Research. Center for Devices and Radiological Health; 2009."},{"key":"10.3233\/JHD-210506_ref19","doi-asserted-by":"crossref","first-page":"257","DOI":"10.5334\/tohm.231","article-title":"The many facets of unawareness in Huntington disease","volume":"4","author":"McCusker","year":"2014","journal-title":"Tremor Other Hyperkinet Mov (N Y)"},{"issue":"4","key":"10.3233\/JHD-210506_ref20","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1212\/WNL.0b013e318260cbe6","article-title":"Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1)","volume":"79","author":"Heatwole","year":"2012","journal-title":"Neurology"},{"issue":"5","key":"10.3233\/JHD-210506_ref21","doi-asserted-by":"crossref","first-page":"577","DOI":"10.1002\/mus.26422","article-title":"The myotonic dystrophy health index: Japanese adaption and validity testing","volume":"59","author":"Mori","year":"2019","journal-title":"Muscle Nerve"},{"issue":"3","key":"10.3233\/JHD-210506_ref22","doi-asserted-by":"crossref","first-page":"233","DOI":"10.3233\/JHD-140115","article-title":"Understanding the outcomes measures used in Huntington disease pharmacological trials: A systematic review","volume":"3","author":"Carlozzi","year":"2014","journal-title":"J Huntingtons Dis"}],"container-title":["Journal of Huntington's Disease"],"original-title":[],"link":[{"URL":"https:\/\/content.iospress.com\/download?id=10.3233\/JHD-210506","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T08:56:06Z","timestamp":1777452966000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.3233\/JHD-210506"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6,3]]},"references-count":19,"journal-issue":{"issue":"2"},"URL":"https:\/\/doi.org\/10.3233\/jhd-210506","relation":{},"ISSN":["1879-6397","1879-6400"],"issn-type":[{"value":"1879-6397","type":"print"},{"value":"1879-6400","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,6,3]]}}}