{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,2]],"date-time":"2026-05-02T01:54:27Z","timestamp":1777686867858,"version":"3.51.4"},"reference-count":16,"publisher":"SAGE Publications","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JHD"],"published-print":{"date-parts":[[2024,7,2]]},"abstract":"<jats:p>Background: Direct-to-participant online reporting facilitates the conduct of clinical research by increasing access and clinically meaningful patient engagement. Objective: We assessed feasibility of online data collection from adults with diagnosed Huntington\u2019s disease (HD) who directly reported their problems and impact in their own words. Methods: Data were collected online from consenting United States residents who self-identified as 1) having been diagnosed with Huntington\u2019s disease, 2) able to ambulate independently, and 3) self-sufficient for most daily needs. Data for this pilot study were collected using the Huntington Study Group myHDstory online research platform. The Huntington Disease Patient Report of Problems (HD-PROP), an open-ended questionnaire, was used to capture verbatim bothersome problems and functional impact. Natural language processing, human-in-the-loop curation of verbatim reports involving clinical and experience experts, and machine learning classified verbatim-reports into clinically meaningful symptoms. Results: All 8 questionnaires in the online pilot study were completed by 345 participants who were 60.9% men, 34.5\u00b19.9 (mean\u00b1SD) years old, and 9.5\u00b18.4 years since HD diagnosis. Racial self-identification was 46.4% Caucasian, 28.7% African American, 15.4% American Indian\/Alaska Native, and 9.5% other. Accuracy of verbatim classification was 99%. Non-motor problems were the most frequently reported symptoms; depression and cognitive impairment were the most common. Conclusions: Online research participation was feasible for a diverse cohort of adults who self-reported an HD diagnosis and predominantly non-motor symptoms related to mood and cognition. Online research tools can help inform what bothers HD patients, identify clinically meaningful outcomes, and facilitate participation by diverse and under-represented populations.<\/jats:p>","DOI":"10.3233\/jhd-231520","type":"journal-article","created":{"date-parts":[[2024,4,30]],"date-time":"2024-04-30T11:41:34Z","timestamp":1714477294000},"page":"237-248","source":"Crossref","is-referenced-by-count":3,"title":["What Huntington\u2019s Disease Patients Say About Their Illness: An Online Direct-to-Participant Pilot Study"],"prefix":"10.1177","volume":"13","author":[{"given":"Karen E.","family":"Anderson","sequence":"first","affiliation":[{"name":"Georgetown University, Washington, DC, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lakshmi","family":"Arbatti","sequence":"additional","affiliation":[{"name":"Grey Matter Technologies\/Modality.AI, San Francisco, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Abhishek","family":"Hosamath","sequence":"additional","affiliation":[{"name":"Grey Matter Technologies\/Modality.AI, San Francisco, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andrew","family":"Feigin","sequence":"additional","affiliation":[{"name":"NYU Langone Health, New York, NY, USA"},{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jody","family":"Goldstein","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elise","family":"Kayson","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brett L.","family":"Kinsler","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lauren","family":"Falanga","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lynn","family":"Denise","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noelle E.","family":"Carlozzi","sequence":"additional","affiliation":[{"name":"University of Michigan, Ann Arbor, Michigan, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Samuel","family":"Frank","sequence":"additional","affiliation":[{"name":"Beth Israel Deaconess Medical Center\/Harvard Medical School, Boston, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katie","family":"Jackson","sequence":"additional","affiliation":[{"name":"Help4HD, Elk Grove, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sandra","family":"Kostyk","sequence":"additional","affiliation":[{"name":"The Ohio State University, Columbus, OH, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer L.","family":"Purks","sequence":"additional","affiliation":[{"name":"University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenneth P.","family":"Serbin","sequence":"additional","affiliation":[{"name":"University of San Diego, San Diego, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shari","family":"Kinel","sequence":"additional","affiliation":[{"name":"Huntington Study Group, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christopher A.","family":"Beck","sequence":"additional","affiliation":[{"name":"University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ira","family":"Shoulson","sequence":"additional","affiliation":[{"name":"Grey Matter Technologies\/Modality.AI, San Francisco, CA, USA"},{"name":"University of Rochester, Rochester, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","reference":[{"key":"10.3233\/JHD-231520_ref1","doi-asserted-by":"crossref","first-page":"509","DOI":"10.3233\/JHD-190380","article-title":"Quality of care for Huntington\u2019s disease in the United States: Findings from a national survey of patients and caregivers","volume":"8","author":"Anderson","year":"2019","journal-title":"J Huntingtons Dis"},{"key":"10.3233\/JHD-231520_ref2","doi-asserted-by":"crossref","first-page":"1957","DOI":"10.3233\/JPD-212636","article-title":"Predictive value of verbatim Parkinson\u2019s disease patient-reported symptoms of postural instability and falling","volume":"11","author":"Javidnia","year":"2021","journal-title":"J Parkinsons Dis"},{"key":"10.3233\/JHD-231520_ref3","doi-asserted-by":"crossref","first-page":"757","DOI":"10.3233\/JPD-225083","article-title":"What patients say: Large-scale analyses of replies to the Parkinson\u2019s Disease Patient Report of Problems (PD-PROP)","volume":"13","author":"Marras","year":"2023","journal-title":"J Parkinsons Dis"},{"key":"10.3233\/JHD-231520_ref4","doi-asserted-by":"crossref","first-page":"1969","DOI":"10.3233\/JPD-223274","article-title":"Longitudinal cohort study of verbatim-reported postural instability symptoms as outcomes for online Parkinson\u2019s disease trials","volume":"12","author":"Shoulson","year":"2022","journal-title":"J Parkinsons Dis"},{"key":"10.3233\/JHD-231520_ref5","doi-asserted-by":"crossref","first-page":"1443","DOI":"10.1007\/s00415-018-8852-5","article-title":"Agreement between clinician-rated versus patient-reported outcomes in Huntington disease","volume":"265","author":"Carlozzi","year":"2018","journal-title":"J Neurology"},{"key":"10.3233\/JHD-231520_ref6","first-page":"136","article-title":"Huntington Study Group","volume":"11","author":"Unified Huntington\u2019s Disease Rating Scale: Reliability and consistency","year":"1996","journal-title":"Movement Dis"},{"key":"10.3233\/JHD-231520_ref8","doi-asserted-by":"crossref","first-page":"494","DOI":"10.1016\/S1474-4422(23)00127-8","article-title":"Safety and efficacy of valbenazine for the treatment of chorea associated with Huntington\u2019s disease (KINECT-HD): A phase 3, randomised, double-blind, placebo-controlled trial","volume":"22","author":"Furr Stimming","year":"2023","journal-title":"Lancet Neurol"},{"key":"10.3233\/JHD-231520_ref9","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1001\/jama.2016.8655","article-title":"Effect of deutetrabenazine on chorea among patients with Huntington disease: A randomized clinical trial","volume":"316","author":"Frank","year":"2016","journal-title":"JAMA"},{"key":"10.3233\/JHD-231520_ref10","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1016\/S1474-4422(18)30391-0","article-title":"Safety and efficacy of pridopidine in patients with Huntington\u2019s disease (PRIDE-HD): A phase 2, randomised, placebo-controlled, multicentre, dose-ranging study","volume":"18","author":"Reilmann","year":"2019","journal-title":"Lancet Neurol"},{"key":"10.3233\/JHD-231520_ref11","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1016\/S1474-4422(14)70262-5","article-title":"Safety, tolerability, and efficacy of PBT2 in Huntington\u2019s disease: A phase 2, randomised, double-blind, placebo-controlled trial","volume":"14","author":"Huntington Study Group Reach2HD Investigators","year":"2015","journal-title":"Lancet Neurol"},{"key":"10.3233\/JHD-231520_ref12","doi-asserted-by":"crossref","first-page":"2183","DOI":"10.1038\/s41591-022-01919-8","article-title":"Pepinemab antibody blockade of SEMA4D in early Huntington\u2019s disease: A randomized, placebo-controlled, phase 2 trial","volume":"28","author":"Feigin","year":"2022","journal-title":"Nature Med"},{"key":"10.3233\/JHD-231520_ref13","doi-asserted-by":"crossref","first-page":"19\u2013023\u201302622\u20138","DOI":"10.1186\/s13023-023-02622-8","article-title":"What we don\u2019t need to prove but need to do in multidisciplinary treatment and care in Huntington\u2019s disease: A position paper","volume":"18","author":"Muhlb\u00e4ck","year":"2023","journal-title":"Orphanet J Rare Dis"},{"key":"10.3233\/JHD-231520_ref14","doi-asserted-by":"crossref","first-page":"667420","DOI":"10.3389\/fneur.2021.667420","article-title":"Enroll-HD: An integrated clinical research platform and worldwide observational study for Huntington\u2019s disease","volume":"12","author":"Sathe","year":"2021","journal-title":"Front Neurol"},{"key":"10.3233\/JHD-231520_ref15","first-page":"212","article-title":"Data analytics from Enroll-HD, a global clinical research platform for Huntington\u2019s disease","volume":"4","author":"Landwehrmeyer","year":"2016","journal-title":"Mov Dis"},{"key":"10.3233\/JHD-231520_ref16","doi-asserted-by":"crossref","first-page":"217","DOI":"10.3233\/JHD-210506","article-title":"The Huntington\u2019s Disease Health Index: Initial evaluation of a disease-specific patient reported outcome measure","volume":"11","author":"Brumfield","year":"2022","journal-title":"J Huntington Dis"},{"key":"10.3233\/JHD-231520_ref17","doi-asserted-by":"crossref","first-page":"632","DOI":"10.1016\/S1474-4422(22)00120-X","article-title":"A biological classification of Huntington\u2019s disease: The Integrated Staging System","volume":"21","author":"Tabrizi","year":"2022","journal-title":"Lancet Neurol"}],"container-title":["Journal of Huntington's Disease"],"original-title":[],"link":[{"URL":"https:\/\/content.iospress.com\/download?id=10.3233\/JHD-231520","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T08:56:24Z","timestamp":1777452984000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.3233\/JHD-231520"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,2]]},"references-count":16,"journal-issue":{"issue":"2"},"URL":"https:\/\/doi.org\/10.3233\/jhd-231520","relation":{},"ISSN":["1879-6397","1879-6400"],"issn-type":[{"value":"1879-6397","type":"print"},{"value":"1879-6400","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,2]]}}}