Lancet
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Primary biliary cholangitis is a chronic, autoimmune, cholestatic disease that mainly affects women aged 40-70 years. Recent epidemiological studies have shown an increasing incidence worldwide despite geographical heterogeneity and a decrease in the female-to-male ratio of those the disease affects. Similar to other autoimmune diseases, primary biliary cholangitis occurs in genetically predisposed individuals upon exposure to environmental triggers, specifically xenobiotics, smoking, and the gut microbiome. ⋯ Improvement of health-related quality of life with better recognition and care of subjective symptoms, such as pruritus and fatigue, is also an important treatment goal. Promising clinical investigations are underway to alleviate these symptoms. Efforts to facilitate better access to medical care and dissemination of current knowledge should enable diagnosis at an earlier stage of primary biliary cholangitis and ensure access to treatments based on risk stratification for all patients.
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Frequentist and Bayesian statistics represent two differing paradigms for the analysis of data. Frequentism became the dominant mode of statistical thinking in medical practice during the 20th century. The advent of modern computing has made Bayesian analysis increasingly accessible, enabling growing use of Bayesian methods in a range of disciplines, including medical research. ⋯ Bayesian analysis combines previous information (represented by a mathematical probability distribution, the prior) with information from the study (the likelihood function) to generate an updated probability distribution (the posterior) representing the information available for clinical decision making. Owing to its fundamentally different conception of probability, Bayesian statistics offers an intuitive, flexible, and informative approach that facilitates the design, analysis, and interpretation of clinical trials. In this Review, we provide a brief account of the philosophical and methodological differences between Bayesian and frequentist approaches and survey the use of Bayesian methods for the design and analysis of clinical research.