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

HD-HRQOL（亨廷顿病生活质量测量）的验证

• 批准号: 8865709
• 负责人: Noelle E. Carlozzi
• 金额: $ 58.8万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-05-15 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8865709
• 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。” NIH 共同基金发起了前所未有的大规模举措，旨在开发与健康相关的生活质量 (HRQOL) 的患者报告结果 (PRO)，用于： 1) 普通人群（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、帕金森病（由NINDS资助）； 3）脊髓损伤（由NCMRR和NINDS资助）； 4) 创伤性脑损伤（由 NIDRR 和 VA RR&D 资助）。最近，NINDS 扩展了这些测量开发工作，将亨廷顿病 (HD) 纳入其中，这是一种致命的、隐匿的、进行性神经退行性疾病，其特征是运动、认知和精神功能异常。 HD 是最具破坏性的神经系统疾病之一，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 中相关的 HRQOL 领域/主题并开发反映这些的项目域/主题。由患有亨廷顿舞蹈症或有患亨廷顿舞蹈症风险的个人、家庭成员和护理人员以及亨廷顿舞蹈症专业人员组成的焦点小组，旨在确定与亨廷顿舞蹈症患者相关的 HRQOL 问题。研究结果表明，亨廷顿舞蹈症患者面临着多种问题（情感、身体、社交、认知和生命终结）。具体而言，调查结果表明，虽然新 PRO 测量的许多结构在该人群中是相关且重要的，但还需要评估这些现有测量系统尚未捕获的 HD 特定 HRQOL 问题（即，最终结果）。生活问题、舞蹈症、言语和吞咽困难）。因此，反映这些 HD 特定结构的项目被开发出来，以补充先前存在的 PRO 计划。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。具体来说，采用最先进的方法，采用经典和现代的测试构建和验证方法（包括项目反应理论和计算机化自适应测试技术）来开发计算机化自适应测试，允许对临床症状进行简单而精确的测量。相关症状和功能限制。此外，本研究还将检查这些措施对随时间变化的敏感性和响应能力。这种新的 HD-QOL 测量系统将允许临床医生通过检测 HD HRQOL 结果中微小但重要的变化来监测患者，使研究人员能够进行更有效的 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沟通他们向供应商提出的 HRQOL 问题。