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 most recent guidelines 1.

Key Considerations

• UDCA should be started as soon as diagnosis is confirmed, even in asymptomatic patients, to reduce bile acid toxicity and slow disease progression 1.
• For those who don't respond adequately to UDCA after 12 months, second-line therapy such as obeticholic acid (Ocaliva) at 5-10 mg daily can be considered 1.
• Symptom management is crucial, with cholestyramine (4 g before meals) recommended for pruritus and fat-soluble vitamin supplements (vitamins A, D, E, and K) for patients with advanced disease 1.
• Regular monitoring of liver function tests every 3-6 months is essential to assess treatment response and identify patients at risk of progressive disease 1.

Lifestyle Modifications

• Patients should avoid alcohol, maintain a healthy weight, and follow a low-fat diet if they have steatorrhea to reduce the risk of disease progression and improve quality of life 1.
• Individualized risk stratification using biochemical response indices is recommended following 1 year of UDCA therapy to identify patients at risk of progressive disease 1.

Advanced Disease

• For advanced disease with decompensated cirrhosis, liver transplantation may ultimately be necessary, and patients should be discussed with a hepatologist linked to a transplant center 1.

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.

• Approved treatment options include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) 3, 4, 5, 6.
• Patients diagnosed with PBC should be offered treatment with 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 OCA at 5 mg per day for patients without cirrhosis or investigational therapy 3, 7, 5, 6.
• Management of common side effects such as pruritus and fatigue is nuanced and includes lifestyle modifications as well as pharmacological approaches 3, 6.

Treatment Options Beyond UDCA

Several studies have investigated treatment options beyond UDCA for patients with PBC.

• Obeticholic acid has been shown to improve liver biochemistries in patients with PBC who are refractory to UDCA 7, 5, 6.
• Fibrates, including fenofibrate and bezafibrate, have also been shown to be beneficial in this population, although more robust studies are needed to confirm these findings 7.
• Other agents, such as methotrexate, colchicine, budesonide, mycophenolate mofetil, and azathioprine, have been studied, but their benefits are marginal and not consistent 7.

Personalized Approach to Treatment

A personalized approach to treatment is necessary, taking into account the individual patient's risk factors and response to treatment.

• On-treatment multifactor-based risk stratification is necessary to identify patients in need of additional therapy 4.
• Complete biochemical normalization may be a preferred goal in selected patients to optimize their outcome 4.
• The use of farnesoid X receptor or peroxisome proliferator-activated receptor agonists, or corticosteroids in highly-selected cases, may be considered to achieve stricter biochemical goals 4.