Computer Adaptive Testing for OA Clinical Trials

描述（由申请人提供）： 需要开发能够改善OA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了解甚少的疾病，也是一种几乎没有有效治疗选择的疾病。OA的治疗进展受到病情进展缓慢、临床表现多样、需要长期随访观察相关症状变化等因素的制约。研究人员的一个重要方法障碍是找到能够确定相关临床结果变化的敏感措施。 本项目的总体目标是应用现代项目反应理论（IRT）和计算机自适应测试（CAT）方法开发临床结果工具，使OA临床试验能够以更高的效率和有效性进行。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项目，以改善现有OA结局工具中的广度、精度和概念清晰度;现场测试修订后的疼痛、僵硬和功能限制问题集，这些问题最终将作为用于开发CAT的项目库;开发用于OA临床试验的原型CAT结果工具，并对其准确性、精确性和有效性进行计算机评估;交叉验证原型CAT的准确性和精确性，并在OA患者样本中对其可接受性和应答负担进行初步测试。 我们将应用IRT和CAT测量的逻辑来评估OA临床干预对疼痛、僵硬和下肢功能的潜在价值，使用从现有工具（如WOMAC）开发的项目库加上项目团队编写的新项目。这些项目池将在从项目团队目前正在进行的研究中抽取的下肢OA患者样本中进行检测。OA研究的CAT结果系统的预期优势是：概念清晰，提高敏感性的临床意义的变化，减少应答负担，增加分数精度比固定长度的形式，消除天花板和地板的影响，监测数据质量在真实的时间，并降低数据收集成本。我们希望在精确度和效率的得分估计的收益上级传统的固定形式的评估和转换结果监测OA临床试验研究。

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.