The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines

Abstract

Primary biliary cholangitis (formerly known as primary biliary cirrhosis, PBC) is an autoimmune liver disease in which a cycle of immune mediated biliary epithelial cell injury, cholestasis and progressive fibrosis can culminate over time in an end-stage biliary cirrhosis. Both genetic and environmental influences are presumed relevant to disease initiation. PBC is most prevalent in women and those over the age of 50, but a spectrum of disease is recognised in adult patients globally; male sex, younger age at onset (<45) and advanced disease at presentation are baseline predictors of poorer outcome. As the disease is increasingly diagnosed through the combination of cholestatic serum liver tests and the presence of antimitochondrial antibodies, most presenting patients are not cirrhotic and the term cholangitis is more accurate. Disease course is frequently accompanied by symptoms that can be burdensome for patients, and management of patients with PBC must address, in a life-long manner, both disease progression and symptom burden. Licensed therapies include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), alongside experimental new and re-purposed agents. Disease management focuses on initiation of UDCA for all patients and risk stratification based on baseline and on-treatment factors, including in particular the response to treatment. Those intolerant of treatment with UDCA or those with high-risk disease as evidenced by UDCA treatment failure (frequently reflected in trial and clinical practice as an alkaline phosphatase >1.67 × upper limit of normal and/or elevated bilirubin) should be considered for second-line therapy, of which OCA is the only currently licensed National Institute for Health and Care Excellence recommended agent. Follow-up of patients is life-long and must address treatment of the disease and management of associated symptoms.

PBC‐10: a short quality of life measure for clinical screening in primary biliary cholangitis

“…We recommend using PBC‐10 in routine clinical setting as a quick symptoms screening questionnaire as well as research or clinical trials to screen for symptoms, as recommended by the international societies . The measure was developed using rigorous statistical and methodological approach using currently recommended guidelines in a large patient cohort. In chronic conditions such as PBC, it is important to measure treatment in terms of impact on quality of life not just survival .…”

“…Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease. An often asymptomatic early phase is typically followed by the progressive development of disease‐related symptoms which can have a significant impact on life quality . Typical PBC‐related symptoms include fatigue, pruritus, dry eyes and mouth, and occasional abdominal and bone pain .…”

“…HRQoL provides insights into how patients perceive their own disease processes, which understandably may be different from the way they are perceived by clinicians. Considering the importance of symptoms and their impact on the quality of life of patients all current guidelines, including those from the British Society of Gastroenterology and the European Association for the Study of the Liver, recommend routine screening for the presence of symptoms in PBC. None of these guidelines, however, suggest how such screening should be achieved in practice, and at present there are no validated tools suitable for screening use in practice.…”

“…An often asymptomatic early phase is typically followed by the progressive development of disease-related symptoms which can have a significant impact on life quality. 1,2 Typical PBC-related symptoms include fatigue, pruritus, dry eyes and mouth, and occasional abdominal and bone pain. 3,4 Fatigue can be profound and persistent, and is typically unrelated to histological stage or activity of the disease.…”

New‐onset primary biliary cholangitis in anti‐mitochondrial antibody‐positive patients may be underestimated without liver biopsy

“…As is well known, the diagnosis of PBC can be established if two of the following three criteria are fulfilled: positive AMA serologic testing, persistently elevated cholestatic liver biochemistry (raised ALP) and/or histological demonstration of nonsuppurative inflammation of interlobular bile ducts. Although the most recent practice guidelines of The British Society of Gastroenterology/UKPBC and the American Association for the Study of Liver Diseases recommended that liver biopsy is not usually required in the diagnosis of PBC, PBC cannot be excluded without liver biopsy [2,3]. If a patient only meets one of two criteria (positive AMA serologic testing and persistently elevated ALP), liver biopsy is required to further substantiate the diagnosis of PBC.…”