What is the initial management for patients with primary biliary cirrhosis or primary sclerosing cholangitis?

Initial Management for Primary Biliary Cirrhosis and Primary Sclerosing Cholangitis

For primary biliary cirrhosis (PBC), ursodeoxycholic acid (UDCA) at 13-15 mg/kg/day is the first-line treatment, while for primary sclerosing cholangitis (PSC), UDCA is not recommended as medical therapy. 1

Primary Biliary Cirrhosis (PBC) Management

First-line Treatment

• UDCA at 13-15 mg/kg/day is recommended as first-line therapy for all patients with PBC 1
• This dosage has been shown to be more effective than lower doses (5-7 mg/kg/day) in improving liver biochemistry and Mayo risk score 2
• Higher doses (23-25 mg/kg/day) do not provide additional benefits over the standard dose 2

Monitoring Response

• Biochemical response should be assessed after 1 year of UDCA therapy 1
• Individualized risk stratification using biochemical response indices should be performed to identify patients at risk of progressive disease 1

Second-line Therapy

• For patients with inadequate response to UDCA or who are intolerant to UDCA, obeticholic acid is indicated 3
• Obeticholic acid is FDA-approved for PBC patients without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension 3
• Important caveat: Obeticholic acid is contraindicated in patients with decompensated cirrhosis, prior decompensation events, or compensated cirrhosis with evidence of portal hypertension due to risk of hepatic decompensation 3

Primary Sclerosing Cholangitis (PSC) Management

Medical Therapy

• UDCA is not recommended for routine treatment of PSC 1
• Corticosteroids and immunosuppressants are not indicated for classic PSC 1
• Exception: Patients with PSC and overlap syndrome (features of autoimmune hepatitis) or IgG4-related sclerosing cholangitis may benefit from corticosteroids 1

Diagnostic Workup

• MRCP should be the principal imaging modality for investigation of suspected PSC 1
• ERCP should be reserved for patients requiring tissue acquisition or therapeutic intervention 1
• Liver biopsy is normally reserved for possible small duct PSC, assessment of suspected overlap variants, or unclear diagnosis 1

Symptom Management

• For pruritus, cholestyramine is first-line treatment, with rifampicin and naltrexone as second-line options 1
• For fatigue, alternative causes should be actively sought and treated 1

Monitoring and Follow-up for Both Conditions

PBC Monitoring

• All patients should be evaluated for symptoms, particularly fatigue and pruritus 1
• Risk assessment for osteoporosis should be performed, with treatment according to national guidelines 1

PSC Monitoring

• Non-invasive investigations (MRCP, dynamic liver MRI, contrast CT) should be performed in patients with new/changing symptoms or evolving laboratory abnormalities 1
• All patients should have risk assessment for osteoporosis 1
• Screening for oesophageal varices should follow international guidelines when there is evidence of cirrhosis and/or portal hypertension 1
• Colonoscopy with biopsies should be performed to identify associated colitis 1

When to Consider Liver Transplantation

• Liver transplantation is the only definitive therapy for advanced PBC and PSC 4, 5
• Patients with bilirubin >50 μmol/L or evidence of decompensated liver disease should be discussed with a transplant center 1
• For PSC, eligibility and referral should be assessed according to national guidelines 1

Common Pitfalls to Avoid

• Avoid using UDCA for PSC despite its effectiveness in PBC 1
• Do not use prognostic models for predicting individual outcomes in PSC patients as no consensus exists regarding the optimal model 1
• Avoid delaying referral for transplant evaluation in patients with advanced disease 4, 5
• Do not overlook screening for associated conditions (inflammatory bowel disease in PSC, autoimmune conditions in PBC) 1

Both conditions require lifelong follow-up due to their unpredictable disease course and serious potential complications 1.