Abstract
BACKGROUND: The aim of the study is to develop a specific patient-reported scale of liver cirrhosis according to the Patient Reported Outcome guidelines of the Food and Drug Administration (FDA), and to examine its capacity to fill gaps in this field. METHODS: A conceptual framework was developed and a preliminary item pool developed through literature review and interviews of 10 patients with liver cirrhosis. With the preliminary items, we performed a pilot survey that included a cognitive test with patients and interviews with experts; the focus was on content and language of the scale. In the item selection stage, seven statistical methods including discrete trends method, discrimination analysis, exploratory factor analysis, Cronbach's α coefficient, correlation coefficient, test-retest reliability, Item-Response Theory were applied to survey data from 200 subjects (150 liver cirrhosis patients and 50 controls). This produced the preliminary Liver Cirrhosis Patient-reported Outcome Measure (LC-PROM). In the next stage, we conducted the survey with 620 subjects (500 patients and 120 controls) to validate reliability, validity and acceptability of this scale. RESULTS: The 55 items and 13 dimensions addressed four domains: physical, psychological, social, and therapeutic. Cronbach's α coefficients were 0.921 for the total scale; the confirmatory factor analysis, t-tests and ANOVA supported scale validity; the model fit index as Root Mean Square Error of Approximation (RMSEA), Root Mean Square Residual (RMR), Normed Fit Index (NFI), Non-Normed Fit Index (NNFI), Comparative Fit Index (CFI) and Incremental Fit Index (IFI) met the criterion generally. The acceptance ratio and response rate indicated good feasibility. CONCLUSIONS: This study developed an accurate and stable patient-reported outcome scale of liver cirrhosis, which is able to evaluate clinical effects effectively, is helpful to patients in recognizing their health condition, and contributes to clinical decision making both for patients and physicians. Additionally, the LC-PROM can perform as an ultimate assessment of medical and health care effects and can inform clinical trials of new drugs for liver cirrhosis.