特发性肺纤维化病证结合模式患者报告结局测评工具的关键技术

Idiopathic pulmonary fibrosis (IPF) is the major and complicated respiratory disease, with multi-factors for clinical outcome indicators, and patient reported outcome instrument is an important component of IPF clinical effect evaluation. However, IPF assessment tools lack of China's cultural background and elements, especially lack of reflecting the advantages of the traditional Chinese medicine (TCM) curative effect at present. At present, the key technologies for developing the instrument are generalizability theory, item response theory, computerized adaptive testing and minimal clinically important differences. The following study will be carried out, to begin with, according to the standard of international scale research methods, summarize and concise the TCM concept of lung, especially ancient and modern literatures for IPF. Then propose Theoretical model and the conceptual framework based on the mode of combination of disease and TCM syndrome, and next selecting and establishing the item bank. In addition, adopting the modem test theory, such as generalizability theory, item response theory and computerized adaptive testing to evaluate the subjects' ability and the difficulty of the items, evaluate the testing time and the calculation amount, accurate individualized evaluation of the reliability, validity, responsiveness and acceptability, estimate the measuring accuracy and comprehensiveness. Finally establish the specific patient reported outcome scale for IPF patients (IPF-PRO). What’s more, assess the practicability and maneuverability of IPF-PRO by determining the minimal clinically important differences through the clinical trials, and provide the quantitative basis and objective criteria for TCM clinical efficacy evaluation. The established methods will provide the inspirations and methods for other chronic disease researches.

特发性肺纤维化（IPF）为重大疑难疾病，临床结局指标具有多维属性，IPF测评工具是其疗效评价的重要部分。目前IPF测评工具缺乏中国文化背景和元素，尤其缺少反映中医疗效优势。概化理论、项目反应理论和计算机自适测试及最小差异值是研制评价测评工具的关键技术。本研究按照规范的量表学国际研制方法流程，总结和凝练中医肺系病理论，提出IPF测评工具的病证结合理论模型与概念框架；筛选条目及条目池，并采用概化理论、项目反应理论和计算机自适应测试等关键技术，评价被试者的能力和条目难度，计算测试时间，做出精确的信度、效度与反映度评价，形成IPF患者报告结局量表。进一步基于随机对照试验，采用锚定法和分布法估算量表的临床最小差异值，评价IPF测评工具的实用性和可操作性，为IPF临床诊疗决策及疗效评价提供量化依据和客观标准。所建立的测评工具研制与评价关键技术将为其他疾病的研究提供可借鉴的思路与方法。

特发性肺纤维化（IPF）为重大疑难疾病，临床结局指标具有多维属性，IPF测评工具是其疗效评价的重要部分。目前IPF测评工具缺乏中国文化背景和元素，尤其缺少反映中医疗效优势。概化理论、项目反应理论和计算机自适测试及最小差异值是研制评价测评工具的关键技术。本研究在分析IPF测评工具现状的基础上，借鉴前期慢性阻塞性肺疾病患者报告结局量表修订版的研制方法，按照规范的量表学国际研制方法流程，在专家小组的指导下，总结和凝练中医肺系病理论，提出了病证结合IPF患者报告结局量表（IPF-PRO）理论模型与概念框架；筛选条目及条目池，并采用经典测量理论、概化理论、项目反应理论等关键技术，评价被试者的能力和条目难度，计算测试时间，综合评价IPF-PRO信度、效度与反映度评价，形成的IPF-PRO具有较好的测量特征。进一步基于文献方法、锚定法和分布法估算IPF-PRO的临床最小差异值，可为IPF临床诊疗决策及疗效评价提供量化工具和评价标准。所建立的测评工具研制与评价关键技术将为其他呼吸疾病的研究提供可借鉴的思路与方法。

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