What is the primary treatment and management approach for a patient diagnosed with primary biliary cholangitis (PBC)?

Primary Biliary Cholangitis: Patient Information and Management

What is Primary Biliary Cholangitis?

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease where your immune system mistakenly attacks the small bile ducts in your liver, causing progressive damage that can eventually lead to cirrhosis if left untreated. 1, 2

First-Line Treatment: You Must Start Ursodeoxycholic Acid (UDCA)

All patients diagnosed with PBC should immediately begin ursodeoxycholic acid (UDCA) at 13-15 mg/kg/day, which must be continued lifelong. 1, 3

• UDCA is the cornerstone of PBC treatment and improves liver biochemistry, delays progression to liver failure, prolongs transplant-free survival, and reduces long-term risk of death. 1
• This medication is safe during pregnancy and breastfeeding, so you should continue taking it even if you become pregnant. 1
• UDCA reduces the likelihood of needing a liver transplant or dying from liver disease, particularly in patients with moderate to severe disease. 3
• Take UDCA with or without food. 4

Monitoring Your Response to Treatment

After 1 year of UDCA treatment, your doctor will assess whether the medication is working adequately using blood tests and risk stratification tools. 1, 3

• Your doctor will check alkaline phosphatase (ALP), bilirubin, and other liver enzymes to determine if you're responding well to UDCA. 1, 3
• If your ALP remains elevated above 1.5 times the upper limit of normal after 12 months of UDCA, you are considered an inadequate responder and need additional treatment. 5
• Even if your ALP is between 1.1-1.5 times normal (considered "adequate response"), you may still benefit from more aggressive treatment if you have advanced liver scarring or are younger than 62 years old. 5
• Your doctor should evaluate you annually for symptoms like fatigue and itching, and assess your risk for osteoporosis. 6, 3

Second-Line Treatment: Obeticholic Acid

If you don't respond adequately to UDCA after 1 year, or cannot tolerate UDCA, obeticholic acid (OCA) should be added to your treatment regimen. 1, 3

• OCA is started at 5 mg once daily for 3 months, then increased to 10 mg once daily if you're tolerating it well and need better biochemical control. 4
• Critical warning: OCA is absolutely contraindicated if you have decompensated cirrhosis (Child-Pugh B or C), a history of liver decompensation, or compensated cirrhosis with signs of portal hypertension (such as ascites, varices, or low platelet counts). 4
• Your doctor must closely monitor you while on OCA for any signs of worsening liver function, as hepatic decompensation and failure have been reported, sometimes requiring liver transplantation. 4

Managing Itching (Pruritus)

Itching is one of the most common and bothersome symptoms in PBC, and unfortunately UDCA does not significantly improve it. 3

For itching, your doctor should start with cholestyramine (4-8 g/day) or colestipol (5-10 g/day) as first-line treatment, given at least 4 hours apart from UDCA. 1

• If cholestyramine doesn't work, rifampicin (300-600 mg daily) is the second-line option. 1, 3
• Other options include sertraline (an antidepressant that helps cholestatic itching), naltrexone or nalmefene (opioid antagonists). 3
• If you're taking OCA and develop intolerable itching, your doctor may add an antihistamine, reduce the OCA dose, or temporarily stop it. 4

Managing Fatigue

There is no consistently effective treatment for fatigue in PBC. 3

• Various medications have been studied (oral antioxidants, ondansetron, fluvoxamine, fluoxetine) but none have shown conclusive benefit. 3
• Fatigue management focuses on lifestyle modifications and treating other contributing factors like thyroid dysfunction or anemia. 3

Lifelong Monitoring and Follow-Up

You will need lifelong follow-up because PBC has an unpredictable course and can develop serious complications. 1, 6

• Regular blood tests to monitor liver function are essential. 6, 3
• If you develop cirrhosis, you'll need screening for esophageal varices (enlarged veins in the esophagus) according to standard guidelines. 6
• Abdominal ultrasound should be performed at baseline to exclude other causes of liver disease. 3
• Bone density testing should be done within 5 years of diagnosis due to increased osteoporosis risk. 3

When Liver Transplantation May Be Needed

Both you and your doctor should be aware that PBC is a well-recognized indication for liver transplantation if the disease progresses despite treatment. 1, 6

• If your bilirubin levels rise significantly or you develop signs of decompensated liver disease (ascites, confusion, bleeding), you should be referred to a transplant center. 6
• After transplantation, UDCA should be continued at 10-15 mg/kg/day for life to prevent disease recurrence and improve long-term survival. 3

Important Medication Interactions

• If you take warfarin (a blood thinner), OCA may decrease its effectiveness, so your INR needs closer monitoring. 4
• If you take bile acid binding resins for itching, take UDCA at least 4 hours before or after the resin. 4
• Avoid cyclosporine with OCA if possible, as it can increase liver enzyme abnormalities. 4

Common Pitfalls to Avoid

• Never confuse PBC with primary sclerosing cholangitis (PSC)—they are completely different diseases with opposite treatment approaches. UDCA is mandatory for PBC but not recommended for PSC. 7, 1
• Don't delay starting UDCA once diagnosed—early treatment slows disease progression. 3
• Don't stop UDCA even if you feel well—this is a lifelong medication. 1
• If you have cirrhosis, make absolutely certain your doctor confirms you don't have portal hypertension before starting OCA, as this can be dangerous. 4