Abstract：
UNASSIGNED: Direct-to-participant online reporting facilitates the conduct of clinical research by increasing access and clinically meaningful patient engagement.
UNASSIGNED: We assessed feasibility of online data collection from adults with diagnosed Huntington\'s disease (HD) who directly reported their problems and impact in their own words.
UNASSIGNED: Data were collected online from consenting United States residents who self-identified as 1) having been diagnosed with Huntington\'s 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.
UNASSIGNED: All 8 questionnaires in the online pilot study were completed by 345 participants who were 60.9% men, 34.5±9.9 (mean±SD) years old, and 9.5±8.4 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.
UNASSIGNED: 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.
UNASSIGNED: We assessed feasibility of online data collection from adults with diagnosed Huntington\'s disease (HD) who directly reported their problems and impact in their own words.
UNASSIGNED: Data were collected online from consenting United States residents who self-identified as 1) having been diagnosed with Huntington\'s 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.
UNASSIGNED: All 8 questionnaires in the online pilot study were completed by 345 participants who were 60.9% men, 34.5±9.9 (mean±SD) years old, and 9.5±8.4 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.
UNASSIGNED: 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.
摘要：
■直接面向参与者的在线报告通过增加访问和临床上有意义的患者参与来促进临床研究的进行。
我们评估了从患有亨廷顿病（HD）的成年人那里收集在线数据的可行性，这些成年人用自己的话直接报告了他们的问题和影响。
■数据是从同意的美国居民那里收集的，这些居民自称为1）被诊断出患有亨廷顿病，2）能够独立行走，3)满足大多数日常需求。这项试点研究的数据是使用亨廷顿研究组myHDstory在线研究平台收集的。亨廷顿病患者问题报告（HD-PROP），一份开放式问卷，被用来逐字捕捉麻烦的问题和功能影响。自然语言处理,涉及临床和经验专家的逐字记录报告，机器学习将逐字记录分类为有临床意义的症状。
在线试点研究中的所有8份问卷均由345名参与者完成，其中60.9%为男性，34.5±9.9（平均值±SD）岁，自HD诊断后9.5±8.4年。种族自我认同是46.4％的高加索人，28.7%的非洲裔美国人，15.4%美洲印第安人/阿拉斯加原住民，其他9.5%。逐字分类的准确率为99%。非运动问题是最常见的症状；抑郁症和认知障碍是最常见的。
■在线研究对于自我报告HD诊断且主要是与情绪和认知相关的非运动症状的不同成人队列是可行的。在线研究工具可以帮助告知困扰HD患者的问题，确定有临床意义的结果，并促进不同和代表性不足的人口的参与。
我们评估了从患有亨廷顿病（HD）的成年人那里收集在线数据的可行性，这些成年人用自己的话直接报告了他们的问题和影响。
■数据是从同意的美国居民那里收集的，这些居民自称为1）被诊断出患有亨廷顿病，2）能够独立行走，3)满足大多数日常需求。这项试点研究的数据是使用亨廷顿研究组myHDstory在线研究平台收集的。亨廷顿病患者问题报告（HD-PROP），一份开放式问卷，被用来逐字捕捉麻烦的问题和功能影响。自然语言处理,涉及临床和经验专家的逐字记录报告，机器学习将逐字记录分类为有临床意义的症状。
在线试点研究中的所有8份问卷均由345名参与者完成，其中60.9%为男性，34.5±9.9（平均值±SD）岁，自HD诊断后9.5±8.4年。种族自我认同是46.4％的高加索人，28.7%的非洲裔美国人，15.4%美洲印第安人/阿拉斯加原住民，其他9.5%。逐字分类的准确率为99%。非运动问题是最常见的症状；抑郁症和认知障碍是最常见的。
■在线研究对于自我报告HD诊断且主要是与情绪和认知相关的非运动症状的不同成人队列是可行的。在线研究工具可以帮助告知困扰HD患者的问题，确定有临床意义的结果，并促进不同和代表性不足的人口的参与。
