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; 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普通基金认识到“迫切需要更好地量化临床上重要的症状和结果，包括疼痛，疲劳和生活质量”。 NIH共同基金已导致了史无前例的大规模计划，以开发患者报告的结果（PRO）（PROS），用于健康相关的生活质量（HRQOL），用于：1）一般人群（由NIH资助）； 2）成人癫痫，中风，肌萎缩性侧面硬化症，多发性硬化症和帕金森氏病（由Ninds资助）； 3）脊髓损伤（由NCMRR和Ninds资助）； 4）脑外伤（由NIDRR和VA RR＆D资助）。最近，NINDS扩展了这些测量开发工作，以包括亨廷顿疾病（HD） - 一种致命，阴险，进行性神经退行性疾病，其特征是运动，认知和精神病功能异常。 HD是最具破坏性的神经系统疾病之一，因为症状逐渐出现并恶化，直到最终导致死亡。开发一种特定于高清的措施，称为“ HD QOL”，复制的最先进的定性方法（在上述PROS项目中使用），以识别高清中相关的HRQOL域/主题，并开发反映这些领域/主题的项目。由HD，家庭成员和护理人员和高清专业人员组成的个人或处于危险中的个人，以确定与HD相关的HRQOL问题。调查结果表明，面临高清个人的各种问题（情感，身体，社会，认知和生命的终结）。具体而言，调查结果表明，尽管新专业人士衡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（即生命终结问题，兄弟，唱片，语音和吞咽困难）尚未捕获的HD特异性HRQOL问题，这些问题尚未捕获。结果，开发了反映这些高清特定构建体的项目，以补充已经存在的Pro计划。拟议的项目将验证HD-QOL和其他相关的，新开发的专业人士在多种HD个体样本中。具体而言，采用经典和当代测试结构和验证方法的最先进方法（包括项目响应理论和计算机自适应测试技术）将用于开发计算机化的自适应测试，以简要介绍临床相关症状和功能限制的简短测量。此外，这项研究将检查这些措施对随着时间的变化的敏感性和反应性。这个新的HD-QOL测量系统将使临床医生能够通过检测高清HRQOL结果的少量但重要的变化来监测患者，从而使研究人员能够进行更有效的HD临床试验（高度敏感的措施需要更少的研究参与者），并允许患者更有效地向其提供者传达HRQOL问题。