Quality of Life for SCI Clinical Trials: Development of the SCI-QOL

描述(由申请人提供)：美国国立卫生研究院路线图已经认识到有效结果评估在临床研究中的重要性，指出：“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。通过这一努力，将开发和测试新技术，以衡量这些自我报告的健康状态和结果，涉及广泛的疾病和疾病严重程度。”尽管呼吁采取行动，但专门为脊髓损伤人群开发的与健康相关的生活质量(HRQOL)指标仍然很少。在SCI临床试验中使用的HRQOL测量往往是通用的工具，包含无关的，有时甚至是攻击性的项目，缺乏临床实用价值。创伤性脊髓损伤会导致一系列突发性和破坏性的生理变化、继发性并发症以及社会和心理功能受损，所有这些都会显著影响HRQOL。为了准确测量临床和研究结果，需要一种有效的SCI特有的生活质量测量方法。本研究的目标是：1)构建针对脊髓损伤患者的具有临床意义的卫生保健相关生活质量(HRQOL)测量量表；2)确保得到的量表与“神经生活质量”测量系统兼容，以确保在临床试验中被现场接受和实用；3)以脊髓损伤患者为样本，实地测试针对脊髓损伤的和通用的神经生活质量题库；4)利用项目反应理论(IRT)方法来提炼和校准题库，以供临床试验中的脊髓损伤人群使用。5)开发用于题库管理的计算机化自适应测试(CAT)程序，并在新的SCI患者样本中对CAT程序进行现场测试。SCI-QOL量表将采用最先进的方法，采用经典和当代的测试构建和验证方法。来自全国各地的五个不同的康复机构，包括四个SCI模型系统，将参与这一新的HRQOL衡量标准的开发，以确保足够的敏感性和特异性。该仪器将被开发为一种CAT，它将在许多情况下实用和灵活地进行管理。最后，这项拟议的研究将通过开发一种有意义的、有效的和简明的HRQOL测量来填补一个重要的空白，用于SCI临床试验的结果测量，并将与NINDS神经生活质量测量相结合。

DESCRIPTION (provided by applicant): The NIH Roadmap has recognized the importance of valid outcomes assessment in clinical research, stating: "There is a pressing need to better quantify clinically important symptoms and outcomes, including pain, fatigue, and quality of life. Through this effort, new technologies will be developed and tested to measure these self-reported health states and outcomes across a wide range of illnesses and disease severities." Despite this call for action, there is a paucity of health-related quality of life (HRQOL) measures that have been developed specifically for use with SCI populations. HRQOL measures used in SCI clinical trials tend to be generic instruments, containing irrelevant and sometimes offensive items which lack clinical utility. Traumatic SCI results in a sudden and devastating constellation of physiological changes, secondary complications, and impaired social and psychological function, all of which significantly impact HRQOL. A valid SCI-specific measure of QOL is needed to accurately measure both clinical and research outcomes. The current study aims to: 1) Construct a clinically relevant helath-related quality of life (HRQOL) measurement scale targeted towards individuals with SCI, 2) Ensure that the resulting scale is compatible with the "Neuro-QOL" measurement system to ensure acceptance by the field and utility in clinical trials, 3) Field Test the SCI-targeted and generic Neuro-QOL item banks with a sample of SCI patients, 4) Utilize Item Response Theory (IRT) methodology to refine and calibrate item banks for use with an SCI population in clinical trials, and 5) Develop a computerized adaptive testing (CAT) program for administration of the item banks and field test the CAT program in a new sample of SCI patients. The SCI-QOL scale will be developed using a state-of-the-art approach, employing both classical and contemporary methods of test construction and validation. Five different rehabilitation facilities from across the nation, including four Model SCI Systems, will participate in the development of this new HRQOL measure to ensure adequate sensitivity and specificity. The instrument will be developed as a CAT that will be practical and flexible for administration in many settings. Finally, the proposed study will fill a significant void by developing a meaningful, valid, and concise measure of HRQOL for use in outcome measurement of SCI clinical trials and will be integrated with the NINDS Neuro-QOL measure.