Supplement III to Validation of the HD-HRQOL (Huntington disease quality of life measure)

HD-HRQOL（亨廷顿病生活质量测量）验证的补充 III

• 批准号: 9272128
• 负责人: Noelle E. Carlozzi
• 金额: $ 3.35万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-30 至 2018-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9272128
• 关键词: Accounting; Adult; Affect; Amyotrophic Lateral Sclerosis; Area; Behavioral; Cause of Death; Child; Chorea; Chronic Disease; Clinic Visits; Clinical; Clinical Research; Clinical Trials; Cognitive; Common Data Element; Communities; Complement; Deglutition; Development; Disease; Effectiveness; Emotional; Epilepsy; Family Caregiver; Family member; Fatigue; Focus Groups; Foundations; Funding; General Population; Generic Drugs; Genes; Health; Hereditary Disease; Huntington Disease; Individual; Information Systems; Intervention; Link; Measurement; Measures; Methodology; Methods; Motor; Multiple Sclerosis; Muscle; National Institute of Neurological Disorders and Stroke; Needs Assessment; Neurodegenerative Disorders; Outcome; Outcome Measure; Pain; Parkinson Disease; Participant; Patient Monitoring; Patient Outcomes Assessments; Patient Self-Report; Patients; Persons; Physicians; Policies; Population; Provider; Publishing; Quality of life; Research; Research Personnel; Respondent; Risk; SF-36; Sampling; Speech; Spinal cord injury; Stroke; Symptoms; System; Technology; Testing; Time; Traumatic Brain Injury; United States National Institutes of Health; Validation; Work; base; clinical practice; clinically relevant; computerized; design; effective therapy; end of life; functional status; health related quality of life; improved; nervous system disorder; response; social; theories; tool; treatment center; user-friendly

DESCRIPTION (provided by applicant): The NIH Common Fund recognizes a "pressing need to better quantify clinically important symptoms and outcomes, including pain, fatigue, and quality of life." The NIH Common Fund has led to an unprecedented number of large-scale initiatives to develop patient reported outcomes (PROs) for health-related quality of life (HRQOL) for use in: 1) the general population (funded by NIH); 2) adult epilepsy, stroke, amyotrophic lateral sclerosis, multiple sclerosis, and Parkinson's disease (funded by NINDS); 3) spinal cord injury (funded by NCMRR and NINDS); and 4) traumatic brain injury (funded by NIDRR and the VA RR&D). More recently, the NINDS has extended these measurement development efforts to include Huntington disease (HD)-a fatal, insidious, progressive neurodegenerative disease characterized by abnormalities in motor, cognitive, and psychiatric functions. HD is one of the more devastating neurological diseases, as symptoms gradually appear and worsen until eventually causing death. The effort to develop an HD-specific measure, called the "HD- QOL," replicated state-of-the art qualitative methodology (utilized in the aforementioned PROs projects) to identify relevant HRQOL domains/themes in HD and develop items that reflect these domains/themes. Focus groups consisting of individuals with or at-risk for HD, family members and caregivers, and HD professionals were conducted to identify issues of HRQOL that were relevant for individuals with HD. Findings indicated the diverse issues (emotional, physical, social, cognitive and end of life) that confront individuals with HD. Specifically, findings demonstrated that while many of the constructs measured by the new PROs are relevant and important in this population, there is also a need for assessment of HD-specific HRQOL issues that are not already captured by these existing measurement systems (i.e., end of life issues, chorea, and speech and swallowing difficulties). As a result, items reflecting these HD-specific constructs were developed to complement the preexisting PRO initiatives. The proposed project will validate the HD-QOL and other relevant, newly developed PROs in a diverse sample of individuals with HD. Specifically, a state-of-the-art approach employing both classical and contemporary methods of test construction and validation - including Item Response Theory and computerized adaptive testing technology - will be used to develop a computerized adaptive test that permits brief and precise measurement of clinically relevant symptoms and functional limitations. In addition, this study will examine these measures' sensitivity and responsiveness to change over time. This new HD-QOL measurement system will allow clinicians to monitor patients by detecting small but important changes in HD HRQOL outcomes, allow researchers to conduct more efficient HD clinical trials (highly sensitive measures require fewer study participants), and allow patients to more effectively communicate their HRQOL concerns to their providers.

描述（由申请人提供）：NIH共同基金认识到“迫切需要更好地量化临床重要症状和结果，包括疼痛，疲劳和生活质量。“美国国立卫生研究院共同基金导致了前所未有的大规模倡议，以制定患者报告的结果（PRO），用于健康相关的生活质量（HRQOL）：1）一般人群（由NIH资助）; 2）成人癫痫、中风、肌萎缩侧索硬化症、多发性硬化症和帕金森病（由NINDS资助）; 3）脊髓损伤（由NCMRR和NINDS资助）;和4）创伤性脑损伤（由NIDRR和VA RR&D资助）。最近，NINDS已经将这些测量开发工作扩展到包括亨廷顿病（HD）-一种致命的、隐匿的、进行性神经退行性疾病，其特征在于运动、认知和精神功能的异常。HD是一种更具破坏性的神经系统疾病，症状逐渐出现并恶化，直至最终导致死亡。开发HD特定测量（称为“HD-QOL”）的努力复制了最先进的定性方法（在上述PRO项目中使用），以确定HD中的相关HRQOL领域/主题，并开发反映这些领域/主题的项目。由HD患者或有HD风险的患者、家庭成员和护理人员以及HD专业人员组成的焦点小组进行了研究，以确定与HD患者相关的HRQOL问题。研究结果表明，HD患者面临的各种问题（情感，身体，社会，认知和生命终结）。具体而言，研究结果表明，虽然新PRO测量的许多结构在该人群中具有相关性和重要性，但还需要评估这些现有测量系统尚未捕获的HD特异性HRQOL问题（即，临终问题、舞蹈病、言语和吞咽困难）。因此，开发了反映这些HD特定结构的项目，以补充先前存在的PRO倡议。拟议的项目将在不同的HD患者样本中验证HD-QOL和其他相关的新开发的PRO。具体而言，一个国家的最先进的方法，采用经典和当代的测试建设和验证的方法-包括项目反应理论和计算机化的自适应测试技术-将被用来开发一个计算机化的自适应测试，允许临床相关的症状和功能限制的简短和精确的测量。此外，本研究将检查这些措施的敏感性和反应能力随时间的变化。这种新的HD-QOL测量系统将允许临床医生通过检测HD HRQOL结局中微小但重要的变化来监测患者，允许研究人员进行更有效的HD临床试验（高度敏感的测量需要更少的研究参与者），并允许患者更有效地将他们的HRQOL问题传达给他们的提供者。

项目成果

Classification of Manifest Huntington Disease using Vowel Distortion Measures.

• DOI: 10.21437/interspeech.2020-2724
• 发表时间: 2020-10
• 期刊: Interspeech
• 作者: Romana A;Bandon J;Carlozzi N;Roberts A;Provost EM
• 通讯作者: Provost EM