Primary Sclerosing Cholangitis Treatment & Management

Updated: Nov 21, 2019
  • Author: Vikas Khurana, MD, FACP, FACG; Chief Editor: Vinay Kumar Kapoor, MBBS, MS, FRCS, FAMS  more...
  • Print
Treatment

Approach Considerations

No approved or proven therapy exists for primary sclerosing cholangitis (PSC). [1, 3, 32] Pharmacotherapy is aimed at treating symptoms and managing complications. Immunosuppressants, bile salts, chelators (eg, cholestyramine for pruritus), and steroids are used in an attempt to control the disease process but have not shown significant benefit.

Further care is dictated by the stage and degree of progression of the disease. It involves care by a gastroenterologist, who must monitor the patient's condition with frequent laboratory tests and maintain vigilance for the many complications of PSC.

Liver transplantation is the only therapy that can alter the eventual outcome, with reported posttransplant survival rates of 85% at 5 years and 70% at 10 years. [6] Suitability for liver transplantation should be determined expeditiously and, if appropriate, offered to the patient. Risk for development of inflammatory bowel disease (IBD) persists even after transplantation.

Endoscopic or percutaneous transhepatic dilation of dominant strictures, with or without stenting, has been shown to alleviate cholestasis and to improve laboratory test results; however, it is not currently believed to affect disease progression. Ruling out malignancy in these strictures is difficult.

Ursodeoxycholic acid studies

Use of ursodeoxycholic acid (UDCA) is debatable and controversial. The American Association for the Study of Liver Diseases (AASLD) does not recommend UDCA for as a routine chemopreventive agent to reduce the risk of colorectal dysplasia in patients with PSC and ulcerative colitis [33] ; the European Association for the Study of the Liver (EASL) recommends oral UDCA (13-15 mg/kg/day) as first-line pharmacotherapy in all patients with PSC, often continued for life. The dosing regimen for UDCA is also under debate, including whether a low dose (15-20 mg/kg/day) or high dose (30 mg/kg/day) is optimal.

UDCA has been shown to improve the liver function profile in some patients and, in conjunction with endoscopic dilation, has shown a survival benefit in some studies.

In a study of adult patients with PSC who were given 28-30 mg/kg/day of UDCA versus placebo, Lindor et al reported that the patients treated with UDCA had a greater decrease in serum liver test results but a higher risk of serious adverse events (ie, varices, cholangiocarcinoma, liver transplantation, and death). The risk of a primary endpoint was 2.3-fold greater for the UDCA-treated group than those in the placebo group and 2.1-fold greater risk for death, transplantation, or minimal listing criteria. [34]

Shi et al performed a meta-analysis of randomized, controlled trials comparing UDCA with placebo or no treatment and found that UDCA improved liver biochemistry study results and that there were trends in histologic and cholangiographic improvement. [35] However, the study also showed no improvement for pruritus and fatigue, liver transplantation, or incidence of death. [35]

Next:

Liver Transplantation

Indications for liver transplantation in patients with primary sclerosing cholangitis (PSC) include variceal bleeding or portal gastropathy, intractable ascites, recurrent cholangitis, progressive muscle wasting, hepatic encephalopathy, and poor quality of life. Survival rates have been shown to be 93.7% at 1 year, 92.2% at 2 years, 86.4% at 5 years, and 69.8% at 10 years. However, PSC recurs in 15-20% of patients after liver transplantation. [10, 36]

In addition to liver transplantation, procedures include restorative proctocolectomy (RPC) with ileal pouch anal anastomosis (IPAA) for patients with ulcerative colitis and biliary reconstructive procedures. [37] Note that proctocolectomy in patients with both PSC and ulcerative colitis has no effect on the course of PSC.

Posttransplantation complications include recurrence of PSC, worsening of inflammatory bowel disease (IBD) activity, and de novo IBD occurrence. [6]

Previous
Next:

Diet and Activity

Patients with steatorrhea are encouraged to include medium-chain triglycerides in their diet. Fat-soluble vitamin deficiency correction should be attempted by supplementation. Oral supplementation is necessary if associated pancreatic enzyme deficiency is present. Calcium supplementation for bone disease may also be needed.

Physical activity should not be restricted; however, in patients with osteoporosis, the possibility of fractures should temper the type of activity allowed.

Previous
Next:

Consultations

A gastroenterologist must be consulted. When needed, surgical consultation should be initiated by the gastroenterologist and when liver transplantation is offered. An endocrinologist may be consulted for management of bone disease.

Previous
Next:

Long-Term Monitoring

Nearly 50% of deaths in patients with primary sclerosing cholangitis (PSC) are due to cancer. [38] All patients with PSC should undergo surveillance for malignancy. Such monitoring includes the following:

  • Colonoscopy and biopsy to rule out inflammatory bowel disease (IBD)
  • Annual or biannual colonoscopy to rule out colorectal carcinoma in patients with PSC and IBD, even after liver transplantation
  • Serum CA 19-9 levels every 6-12 months for surveillance of cholangiocarcinoma
  • Annual ultrasonography to monitor for gall bladder cancer
Previous