PBC Good Care Guide

Designed by PBC Ireland, the PBC Good Care Guide explains what good medical care for Primary Biliary Cholangitis should look like for most PBC patients, from diagnosis through long-term monitoring. It helps patients understand the typical steps in PBC care, including treatment, monitoring, and symptom management. By understanding this pathway, patients can have more informed conversations with their healthcare team and feel more confident they are receiving appropriate care.

Early and Accurate Diagnosis

PBC is often first suspected when routine blood tests show a persistent elevation of alkaline phosphatase (ALP). Early recognition and investigation are important so that treatment can begin promptly. Typical investigations include repeat blood tests confirming elevated ALP, antimitochondrial antibodies (AMA), a liver autoimmune antibody panel, and liver ultrasound to exclude bile duct obstruction. In most cases, PBC can be diagnosed when blood tests show a persistent rise in alkaline phosphatase (ALP) and antimitochondrial antibodies (AMA) are present, after other causes of bile duct problems have been ruled out. A liver biopsy is usually not required unless the diagnosis is unclear.

Immediate First-Line Treatment

Once Primary Biliary Cholangitis (PBC) is diagnosed, treatment should begin promptly. All patients should usually start ursodeoxycholic acid (UDCA / Urso). The recommended dose is 13-15 mg/kg/day. For example, a 70 kg patient would usually need approximately 900-1000 mg/day. Treatment should begin as soon as the diagnosis is confirmed, as early therapy significantly improves long-term outcomes and reduces the risk of disease progression.

Baseline Risk Assessment

At diagnosis, clinicians should assess the stage of liver disease. Typical tools include FibroScan (liver stiffness measurement), platelet count, liver ultrasound, and occasionally liver biopsy if diagnosis or staging is uncertain. The purpose is to assess the presence of fibrosis or cirrhosis, determine the patient's risk of disease progression, and guide the frequency of monitoring and follow-up. Patients with more advanced disease require closer monitoring and specialist follow-up.

Assess Treatment Response

Assessing response to treatment is one of the most important steps in PBC care. After approximately 6-12 months of UDCA therapy, clinicians should evaluate the patient's biochemical response. Key markers include ALP, bilirubin, and ALT / AST. Several validated scoring systems may be used, including the Paris II Criteria and the GLOBE Score. The best outcomes are seen when ALP normalises, or is close to normal, and bilirubin remains within the normal range. Patients who do not achieve an adequate biochemical response should be considered for additional therapy and closer monitoring.

Escalation to Second-Line Therapy

If response to UDCA is insufficient, guidelines recommend considering additional therapy. Treatment options may include fibrates, such as fenofibrate or bezafibrate. Patients with an inadequate biochemical response should be reviewed for additional treatment options and closer monitoring. In routine practice this step may sometimes be missed, which can leave some patients undertreated despite ongoing disease activity.

Ongoing Monitoring

Patients with stable PBC are typically reviewed every 6-12 months. Monitoring usually includes blood tests such as ALP, bilirubin, ALT / AST, albumin, and platelet count, along with liver assessment using fibrosis assessment, for example FibroScan, and liver ultrasound when appropriate. Patients with cirrhosis may require additional surveillance, including regular screening for liver cancer. Regular monitoring helps detect changes in disease activity or progression early, allowing treatment and follow-up to be adjusted when needed.

Symptom Management

Symptom management is an important part of PBC care and can have a significant impact on quality of life. Consultants should address the full range of symptoms experienced by the patient, or refer to the appropriate specialist when additional expertise is required. Common symptoms include fatigue, itching (pruritus), cognitive difficulties ("brain fog"), and dry eyes and dry mouth. Treatments for cholestatic itch may include cholestyramine (Questran), rifampicin, or naltrexone. Fatigue is common in PBC and can be difficult to treat, but it should still be recognised, discussed, and managed where possible.

Prevention of Complications

Long-term care should also focus on preventing and monitoring complications associated with PBC. Key areas include bone health, including screening for osteoporosis and monitoring and supplementation of vitamin D; fat-soluble vitamins, including assessment of vitamins A, D, E, and K in patients with advanced cholestasis; cardiovascular risk, with cholesterol and general cardiovascular risk factors assessed as part of routine long-term care; and associated autoimmune conditions, including thyroid disease and Sjögren's syndrome. Proactive monitoring helps identify complications early and supports comprehensive long-term management of PBC.

Advanced Disease Care

If cirrhosis develops, management becomes similar to that for other chronic liver diseases and requires closer specialist follow-up. Care may include monitoring for portal hypertension, screening for oesophageal varices, and surveillance for hepatocellular carcinoma (liver cancer). In cases of advanced liver disease, patients may be evaluated for liver transplantation. Fortunately, with early diagnosis and effective treatment, most patients with PBC do not progress to this stage.

Patient Education and Self-Management

Good care also involves informed and engaged patients who understand their condition and participate actively in their care. Patients should be supported to understand their diagnosis and how PBC affects the liver, their key blood test results, particularly ALP and bilirubin, whether their treatment is working and what treatment goals are, their UDCA / Urso dose and the importance of taking it consistently, the purpose of monitoring tests and clinic visits, symptoms that should be reported, such as worsening fatigue, itch, or jaundice, and the importance of bone health, general health, and lifestyle factors. Providing clear information and practical tools helps patients communicate more effectively with their healthcare team and participate in decisions about their care, supporting better long-term outcomes.