From the Guidelines
Primary sclerosing cholangitis (PSC) treatment should focus on managing symptoms and complications, with liver transplantation being the definitive treatment for end-stage disease, as recommended by the most recent guidelines 1. The treatment approach for PSC involves a combination of medical and endoscopic interventions.
- Medical management includes the use of ursodeoxycholic acid (UDCA) at 13-15 mg/kg/day to improve liver biochemistry, although its impact on disease progression is still debated 1.
- Symptom management is also crucial, with cholestyramine (4-16 g/day) used to control pruritus, and antibiotics like ciprofloxacin or metronidazole used to treat bacterial cholangitis episodes 1.
- Endoscopic procedures, including ERCP with balloon dilation or stent placement, play a key role in managing dominant strictures, with biliary dilatation preferred over the insertion of biliary stents 1.
- Patients should also receive fat-soluble vitamin supplementation (vitamins A, D, E, K) and calcium/vitamin D for bone health, as poor nutrition and fat-soluble vitamin deficiency are common in advanced PSC 1.
- Regular monitoring with liver function tests every 3-6 months and annual imaging is essential to detect any complications early, including cholangiocarcinoma and colorectal cancer, particularly in patients with concurrent inflammatory bowel disease 1.
- For patients undergoing liver transplantation, there is no specific immunosuppressive regimen or treatment effective in preventing PSC recurrence, and the type of biliary anastomosis does not affect the risk of disease recurrence 1.
From the Research
Treatment Options for Primary Sclerosing Cholangitis
- There are limited options for drug treatment of primary sclerosing cholangitis (PSC) 2, 3, 4, 5.
- Ursodeoxycholic acid (UDCA) has been studied as a potential treatment, but randomized, controlled trials have not shown any improvement in outcomes 2, 6.
- Interventional endoscopy may be used to treat dominant stenoses and cholangitis, although this method is supported by low-level evidence 2.
- Liver transplantation is currently the only life-extending therapeutic approach for eligible patients with end-stage PSC, with a 10-year survival rate above 80% 2, 3, 4, 5.
Management of Complications
- PSC can be complicated by bacterial cholangitis, dominant strictures, gallbladder polyps and adenocarcinoma, cholangiocarcinoma, and colorectal malignancy in patients with inflammatory bowel disease (IBD) 3, 4, 5.
- Management of PSC is mainly focused on treatment of symptoms and addressing complications 3, 4, 5.
- Cholangiocarcinoma is the most common malignancy in PSC, with a cumulative lifetime risk of 10-20% 3.
Disease Progression and Recurrence
- PSC is a progressive disease, and no effective medical therapy has been identified to halt disease progression 2, 3, 4, 5.
- Recurrence of PSC after liver transplantation is observed in up to 20% of patients 4.
- The time from diagnosis to death or liver transplantation is 12 to 18 years, and the risk of death from cancer is 40% to 58% 2.