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.

These guidelines on the management of primary sclerosing cholangitis (PSC) were commissioned by the British Society of Gastroenterology liver section. The guideline writing committee included medical representatives from hepatology and gastroenterology groups as well as patient representatives from PSC Support. The guidelines aim to support general physicians, gastroenterologists and surgeons in managing adults with PSC or those presenting with similar cholangiopathies which may mimic PSC, such as IgG4 sclerosing cholangitis. It also acts as a reference for patients with PSC to help them understand their own management. Quality of evidence is presented using the AGREE II format. Guidance is meant to be used as a reference rather than for rigid protocol-based care as we understand that management of patients often requires individual patient-centred considerations.

ObjectivePatient ownership of disease is vital in rare diseases like primary biliary cholangitis (PBC). This survey of UK members of the PBC foundation aimed to assess patients’ perception of their disease management, focusing on key biomarkers and problematic symptoms.DesignRegistered PBC foundation members were surveyed on their experiences on their most recent clinic visit, covering the type of hospital and clinician and whether biochemical response and symptom burden were discussed, including who initiated these conversations. Respondents were also asked about their willingness to initiate these conversations.ResultsAcross 633 respondents, 42% remembered discussing alkaline phosphatase, the key biochemical response measure, and the majority of discussions were initiated by the healthcare provider. 56% of respondents remembered discussing itch, a key PBC symptom. There was no distinction between the grade of healthcare professional, but both patients and clinicians were significantly more likely to discuss symptoms over disease progression. Reassuringly, 84% of respondents felt willing to initiate conversations about their illness, regardless of the grade of managing clinician.ConclusionsThis work lays a positive foundation for patient education and empowerment projects, likely to improve clinical outcomes. Key aspects of management (biochemical response to treatment and symptom burden) should be emphasised as topics of discussion to both patients and clinicians managing PBC. We suggest a simple cue card to prompt patient-led discussion.

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