肝硬化PRO量表的研制及MCID的确定

发布时间：2018-05-22 17:16

本文选题：肝硬化 + 患者报告结局（PRO）　；参考：《山西医科大学》2014年硕士论文

【摘要】：研究目的：本研究严格遵循美国食品及药物管理局（FDA）规定的基于患者报告结局（patientreport outcomes, PRO）量表的制作原则与流程，从患者角度出发，对量表条目进行编制、筛选及测评，制定出适合我国西医肝硬化患者的PRO量表。并通过计算最小临床意义变化值(minimal clinical important difference，MCID)解决PRO量表分值变化的临床解释问题，使该量表能广泛应用于临床疗效评价和新药临床试验中。研究方法：根据国际PRO量表的研制规范，通过查阅文献、患者访谈及借鉴国内外相关量表，收集和提取肝硬化患者的重要信息，形成概念框架，列出条目池。再经过各个领域的专家和患者的认知测试对条目池进行修正，形成量表初稿。用此量表分别在山西省不同城市的八所不同等级的医院进行小规模的预调查，采用经典测量理论和项目反应理论共7种方法对条目进行筛选并结合专业知识，剔除不符合要求的条目，形成初量表。利用初量表做大规模的正式调查，回收后运用与预调查相同的方法再次对条目进行筛选，形成终量表，并对其进行信度和效度分析。用终量表调查100例患者，计算轻微变化组与未改变组得分均值之差，即最小临床意义变化值。研究结果：1．编制出“肝硬化患者PRO评价量表”。量表共有50个条目，，4个领域，13个维度。分为生理、心理、社会、治疗4个领域，其中生理领域包括腹部症状、皮肤症状、食欲睡眠、认知能力、独立性5个维度；心理领域包括焦虑抑郁、自信心、疾病结局3个维度；社会领域包括社会支持、社会适应2个维度；治疗领域包括满意度、依从性、药物副作用3个维度；2．对终量表进行效度和信度分析。结果显示总量表的克朗巴赫系数为0.921，结构效度显示各项模型拟合指标几乎都达到了参考标准中的要求；3.我们最终确定肝硬化PRO评定量表生理领域、心理领域、社会领域和治疗领域的最小临床意义变化值分别为7.06、4.65、3.02和2.08。研究结论：本研究研制的基于肝硬化患者报告的临床结局评价量表具有较好的信度和效度，并且计算出了最小临床意义变化值，解决了PRO量表分值变化的临床解释问题，充分说明本量表可以作为评价肝硬化疾病临床疗效的工具。
[Abstract]:Objective: the purpose of this study was to follow the principles and procedures of the patient reporting report outcomers (Pros) scale based on the FDAs prescribed by the Food and Drug Administration of the United States. From the patient's point of view, the items of the scale were compiled, screened and evaluated. To develop a suitable PRO scale for patients with liver cirrhosis in western medicine. The minimum clinical important difference value was calculated to solve the problem of clinical interpretation of the change of PRO scale, so that it could be widely used in clinical efficacy evaluation and clinical trials of new drugs. Methods: according to the development standard of international PRO scale, the important information of cirrhotic patients was collected and extracted by consulting literature, interviewing patients and referring to relevant scales at home and abroad to form a conceptual framework and list items pool. After the experts and patients in various fields of cognitive tests to amend the pool of items to form a preliminary draft of the scale. The scale was used to carry out small-scale pre-investigation in eight hospitals of different levels in different cities of Shanxi Province, and seven methods were used to screen items and combine professional knowledge with classical measurement theory and item response theory. Eliminate items that do not meet the requirements and form a preliminary scale. A large-scale formal investigation was made by using the initial scale, and the items were screened again with the same method as the pre-survey after recovery, and the final scale was formed, and the reliability and validity of the final scale were analyzed. A total of 100 patients were investigated with the final scale to calculate the difference of the mean score between the slight change group and the unaltered group, that is, the minimum clinical significance change value. The result of the study was: 1. The PRO evaluation scale for patients with liver cirrhosis was developed. The scale consists of 50 items, 4 domains and 13 dimensions. It is divided into four fields: physiology, psychology, society and treatment, including abdominal symptoms, skin symptoms, appetite and sleep, cognitive ability, independence, anxiety and depression, self-confidence, anxiety and depression, Three dimensions of disease outcome; two dimensions of social support and social adaptation; two dimensions of treatment, including satisfaction, compliance, drug side effects. 2. The validity and reliability of the final scale were analyzed. The results showed that the Krombach coefficient of the total table was 0.921, and the structural validity showed that almost all the model fitting indexes met the requirements of the reference standard. We finally determined the minimum clinical significance of the PRO scale for cirrhotic cirrhosis in the physiological, psychological, social and therapeutic fields, respectively, and the minimum clinical significance values were 7.06-4.65V3.02 and 2.08. Conclusion: the clinical outcome evaluation scale based on the report of cirrhotic patients has good reliability and validity, and the minimum clinical significance change value is calculated, and the clinical interpretation of the change of PRO scale score is solved. It is fully demonstrated that this scale can be used as a tool to evaluate the clinical efficacy of liver cirrhosis.
【学位授予单位】：山西医科大学
【学位级别】：硕士
【学位授予年份】：2014
【分类号】：R575.2

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