Computer Adaptive Testing for OA Clinical Trials

描述(由申请人提供)： 开发能够改善骨性关节炎症状的治疗方法是一个重要的研究目标。骨关节炎仍然是一种知之甚少的疾病，也是一种几乎没有有效治疗选择的疾病。骨性关节炎的治疗进展缓慢，其临床表现异质性，需要长期随访观察相关症状的变化，制约了治疗的发展。研究人员面临的一个重要的方法学障碍是寻找能够确定相关临床结果变化的敏感方法。 这个项目的总体目标是应用当代项目反应理论(IRT)和计算机自适应测试(CAT)方法来开发临床结果工具，使OA临床试验能够更高效和更有效地进行。我们将通过以下方式实现这一目标：为疼痛、僵硬和功能限制量表创建新的条目，以改进现有OA结果工具中的广度、精密度和概念清晰度；现场测试修订的疼痛、僵硬、功能限制问题集，最终将用作CAT开发的项目库；为OA临床试验开发CAT结果工具原型，并对其准确性、精密度和有效性进行计算机评估；以及交叉验证CAT原型的准确性和精密度，并在一组OA患者中进行试点测试其可接受性和反应负担。 我们将应用IRT和CAT测量的逻辑来评估OA临床干预在疼痛、僵硬和下肢功能方面的潜在价值，使用从现有工具(如WOMAC)开发的题库以及项目团队编写的新项目。这些题库将在项目团队目前正在进行的研究中抽取的下肢骨关节炎患者样本中进行测试。对于OA研究，CAT结果系统的预期优势是：概念清晰，对临床上有意义的变化更敏感，减少了受访者的负担，比固定长度的表格提高了评分精度，消除了上限和下限影响，实时监测数据质量，以及较低的数据收集成本。我们期望评分评估的精确度和效率的提高将优于传统的固定评估形式，并在OA临床试验研究中改变结果监测。

DESCRIPTION (provided by applicant): The need to develop treatment approaches that are capable of ameliorating the symptoms of OA is an important research objective. OA remains a condition that is poorly understood, and a condition for which few effective therapeutic options are available. Therapeutic development in OA is constrained by the slow progress of the condition, its heterogeneous clinical manifestations, the need for long-term follow-up to observe changes in relevant symptoms. An important methodological obstacle by researchers is finding sensitive measures able to ascertain changes in relevant clinical outcomes. The overall objective of this project is to apply contemporary Item Response Theory (IRT) and Computer Adaptive Test (CAT) methods to develop clinical outcome instruments that permit OA clinical trials to be done with greater efficiency and effectiveness. We will achieve this objective by: creating new items for pain, stiffness, and functional limitation scales to improve upon the breadth, precision, and conceptual clarity found in existing OA outcome instruments; field testing revised sets of pain, stiffness, functional limitation questions that will eventually serve as the item pool used for the development of a CAT; developing a prototype CAT outcome instruments for OA clinical trials and conduct computer estimates of its accuracy, precision and validity; and cross validating the prototype CAT for accuracy and precision and pilot test its acceptability and respondent burden in a sample of patients with OA. We will apply the logic of IRT and CAT measurement to assess the potential value of OA clinical interventions on pain, stiffness, and lower extremity function, using item pools developed from existing instruments like the WOMAC plus new items written by the project team. These item pools will be tested in samples of individuals with lower extremity OA drawn from studies currently being conducted by the project team. The expected advantages of a CAT outcome system for OA research are: conceptual clarity, improved sensitivity to clinically meaningful change, reduced respondent burden, increased score precision over fixed-length forms, elimination of ceiling and floor effects, monitoring of data quality in real time, and lower data collection costs. We expect the gains in precision and efficiency of score estimation to be superior over traditional fixed forms of assessments and transform outcomes monitoring in OA clinical trials research.