What is the best way to manage Primary Biliary Cholangitis (PBC)?

From the Guidelines

The best way to manage Primary Biliary Cholangitis (PBC) is through a combination of medication therapy and lifestyle modifications, with Ursodeoxycholic acid (UDCA) as the first-line treatment, typically prescribed at 13-15 mg/kg/day divided into 2-4 doses, as recommended by the British Society of Gastroenterology/UK-PBC guidelines 1.

Key Considerations

• UDCA has been demonstrated to exert anticholestatic effects, improve bile flow, and decrease inflammation in the bile ducts, thereby slowing disease progression and potentially delaying the need for liver transplantation 1.
• The treatment should be started as soon as diagnosis is confirmed, and patients should be evaluated for the presence of symptoms, particularly fatigue and itch, to ensure appropriate investigation and treatment 1.
• For patients who don't respond adequately to UDCA after 12 months, obeticholic acid (Ocaliva) at 5-10 mg daily can be added as second-line therapy, although this is not universally recommended and should be considered on a case-by-case basis.

Symptom Management

• Cholestyramine (4 g before meals) can help with pruritus, while fat-soluble vitamin supplements (vitamins A, D, E, and K) are recommended to prevent deficiencies 1.
• Patients should avoid alcohol, maintain a low-sodium diet if they have ascites, and undergo regular monitoring of liver function tests every 3-6 months.

Monitoring and Follow-up

• Bone density scans should be performed every 2-3 years to monitor for osteoporosis, which is common in PBC.
• Patients with advanced disease, as reflected by serum bilirubin exceeding 6 mg/dL or decompensated cirrhosis, should be considered for liver transplantation 1.

Treatment Goals

• The primary goal of treatment is to slow disease progression, improve quality of life, and delay the need for liver transplantation.
• Treatment should be individualized, taking into account the patient's biochemical response to UDCA, symptoms, and overall health status.

From the FDA Drug Label

OCALIVA is indicated for the treatment of adult patients with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA. The recommended dosage of OCALIVA for PBC patients without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, who have not achieved an adequate biochemical response to an appropriate dosage of UDCA for at least 1 year or are intolerant to UDCA follows below: Start with a dosage of 5 mg once daily for the first 3 months. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating OCALIVA, increase to a maximum dosage of 10 mg once daily.

The best way to manage PBC is to use obeticholic acid (OCALIVA) in combination with ursodeoxycholic acid (UDCA) for patients with an inadequate response to UDCA, or as monotherapy for patients unable to tolerate UDCA. The recommended dosage is to start with 5 mg once daily for the first 3 months, and then increase to a maximum of 10 mg once daily if the patient has not achieved an adequate reduction in ALP and/or total bilirubin and is tolerating OCALIVA 2.

From the Research

Management of Primary Biliary Cholangitis (PBC)

The management of PBC involves a combination of pharmacological and lifestyle approaches.

• Patients diagnosed with PBC should be offered treatment with ursodeoxycholic acid (UDCA) at 13-15 mg/kg per day if liver enzymes are elevated 3.
• If patients do not meet the defined criteria of response, adjunctive therapy should be considered, which may include obeticholic acid (OCA) at 5 mg per day for patients without cirrhosis or investigational therapy 3, 4.
• The goal of therapy should be the normalization of ALP and bilirubin below 0.6 the upper limit of normal 4.
• Management of side effects such as pruritus and fatigue is nuanced and includes lifestyle modifications as well as pharmacological approaches 3.

Treatment Options for PBC

Several treatment options are available for PBC, including:

• Ursodeoxycholic acid (UDCA) as first-line treatment 3, 4, 5, 6, 7
• Obeticholic acid (OCA) as second-line treatment for patients with inadequate response to UDCA 3, 4, 6, 7
• Bezafibrate as an alternative option for patients with inadequate response to UDCA 4, 6
• Budesonide for patients with marked portal inflammation 4
• Triple therapy with UDCA, OCA, and bezafibrate for patients showing inadequate response to dual therapy 4

Personalized Approach to PBC Management

A personalized approach is necessary for the management of PBC, taking into account the individual patient's risk of liver disease progression and response to treatment 5.

• On-treatment multifactor-based risk stratification is necessary to identify patients in need of additional therapy 5.
• Complete biochemical normalization may be preferred in selected patients to optimize their outcome 5.