What is the treatment for primary sclerosing cholangitis (PSC)?

Treatment of Primary Sclerosing Cholangitis (PSC)

There is currently no effective medical therapy that alters the natural history of PSC, and liver transplantation remains the only definitive treatment for end-stage disease. 1

Disease Management Approach

Medical Management

• Ursodeoxycholic acid (UDCA):

  • Not recommended for routine treatment of PSC (strong recommendation, moderate quality evidence) 1
  • Despite improving biochemical parameters, UDCA has not been shown to improve survival or disease progression 1

• Immunosuppressive therapy:

  • Not indicated for classic PSC (strong recommendation, high quality evidence) 1
  • Only recommended for patients with PSC-autoimmune hepatitis overlap syndrome 1

Management of Dominant Strictures

Dominant strictures occur in 45-58% of patients during follow-up 1 and require prompt evaluation:

1. Evaluation:

   • Perform ERCP when patients present with increased bilirubin, worsening pruritus, bile duct dilatation, or cholangitis 1
   • Mandatory pathological sampling of suspicious strictures to exclude malignancy 1

2. Treatment:

   • Preferred approach: Endoscopic balloon dilatation without stenting 1
   • Stenting should be reserved for strictures refractory to dilatation due to increased complication risk 1
   • Prophylactic antibiotics should be administered during ERCP 1

3. Alternative approaches:

   • Percutaneous cholangiography with dilatation if endoscopic approach fails 1
   • Surgical therapy only in selected non-cirrhotic patients with refractory strictures 1

Management of Complications

Bacterial Cholangitis

• Treat with antimicrobial therapy plus correction of bile duct obstruction 1
• For recurrent bacterial cholangitis:
  • Consider prophylactic long-term antibiotics 1
  • Evaluate for liver transplantation if refractory 1

Pruritus

• First-line: Cholestyramine or similar bile acid sequestrants 1
• Second-line: Rifampicin and naltrexone 1
• Always evaluate for dominant strictures when pruritus worsens 1

Metabolic Bone Disease

• Perform osteoporosis risk assessment for all PSC patients 1
• Provide calcium and vitamin D supplementation 2
• Consider bisphosphonates in selected cases 2

Nutritional Support

• Low threshold for empirical replacement of fat-soluble vitamins in advanced disease 1
• Consider parenteral vitamin K in patients with severe cholestasis and coagulation defects 2

Surveillance

Cholangiocarcinoma

• No role for routine CA19.9 measurement for surveillance 1
• For suspected cholangiocarcinoma:
  • Refer for specialist multidisciplinary review 1
  • Perform contrast-enhanced cross-sectional imaging 1
  • Obtain histological confirmation via ERCP-guided sampling or other methods 1

Colorectal Cancer

• For patients with PSC and IBD: Annual colonoscopic surveillance from diagnosis of colitis 1
• For patients without IBD: Consider less frequent (5-year) colonoscopy 1

Gallbladder Disease

• Annual ultrasound of gallbladder 1
• If polyps are identified, refer to specialist hepatopancreaticobiliary MDM 1

Hepatocellular Carcinoma

• In cirrhotic patients, follow international guidelines for HCC surveillance 1

Liver Transplantation

• Well-established indication for PSC 1
• Eligibility and referral should follow national guidelines 1
• Provides 10-year survival rates above 80% 3
• Indications include:
  • End-stage liver disease
  • Intractable pruritus
  • Recurrent bacterial cholangitis
  • Suspected early cholangiocarcinoma in selected centers

Clinical Pearls and Pitfalls

• Pitfall: Delaying ERCP in patients with worsening symptoms or laboratory values

  • Solution: Promptly evaluate for dominant strictures when clinical deterioration occurs

• Pitfall: Missing cholangiocarcinoma, which occurs in 10-15% of PSC patients

  • Solution: Mandatory pathological sampling of suspicious strictures during ERCP

• Pitfall: Overuse of biliary stents

  • Solution: Prefer balloon dilatation to stenting for dominant strictures

• Pitfall: Inadequate surveillance for associated malignancies

  • Solution: Implement structured surveillance protocols for cholangiocarcinoma, colorectal cancer, and gallbladder cancer

• Caveat: Disease course is unpredictable and requires lifelong follow-up 1