Opinion statement
Aims of treatment for primary sclerosing cholangitis are as follows: prevention of progression of hepatobiliary disease, reduction of symptoms and consequences of cholestasis (pruritus, osteoporosis), and prevention of complications (colorectal cancer, hepatobiliary cancer). Ursodeoxycholic acid (UDCA) improves biliary secretion and laboratory parameters of cholestasis, but its effects on liver histology and survival are not clear. It reduces the incidence of dysplasias and carcinomas of the colon in patients with colitis and possibly has a beneficial effect on the incidence of bile duct carcinomas. At present, UDCA represents the most promising therapeutic option. Immunosuppressive treatment has not been proven to be effective; it appears to be indicated in the overlap syndrome with autoimmune hepatitis but may be harmful in bacterial cholangitis. Bacterial cholangitis is common in patients with dominant stenoses and requires antibiotic treatment. Endoscopic treatment of dominant stenoses improves cholestasis and prolongs survival in comparison to predicted survival. Pruritus represents a problem in some patients, and cholestyramine represents the first-line treatment. If ineffective, opioid antagonists, rifampin, or ondansetron may be tried. For treatment of osteoporosis and osteopenia, calcium and vitamin D supplementation are recommended, and in selected cases, bisphosphonates may be indicated. In patients with severe cholestasis and coagulation defects, parenteral supplementation of vitamin K may be indicated. During treatment, all patients should be regularly screened for colonic and bile duct carcinomas. Patients with cirrhosis of the liver and its complications are treated accordingly, and in end-stage disease, liver transplantation is indicated.
Similar content being viewed by others
References and Recommended Reading
Chapman RW, Arborgh BA, Rhodes JM, et al.: Primary sclerosing cholangitis: a review of its clinical features, cholangiography, and hepatic histology. Gut 1980, 21:870–877.
Wiesner RH, Grambsch PM, Dickson ER, et al.: Natural history, prognostic factors, and survival analysis. Hepatology 1989, 10:430–436.
Broome U, Olson R, Lööf L, et al.: Natural history and prognostic factors in 305 Swedish patients with primary sclerosing cholangitis. Gut 1996, 38:610–615.
Olsson R, Danielsson A, Järnebrot G, et al.: Prevalence of primary sclerosing cholangitis in patients with ulcerative colitis. Gastroenterology 1991, 100:1319–1323.
Pohl J, Ring A, Stremmel W, Stiehl A: The role of dominant stenoses in bacterial infections of bile ducts in primary sclerosing cholangitis. Eur J Gastroenterol Hepatol 2006, 18:69–74.
Bergquist A, Ekbom A, Olsson R, et al.: Hepatic and extrahepatic malignancies in primary sclerosing cholangitis. J Hepatol 2002, 36:321–327.
Ponsioen CY, Vrouenraets SM, van Milligen de Wit AW, et al.: Value of brush border cytology for dominant strictures in primary sclerosing cholangitis. Endoscopy 1999, 31:305–309.
Hultcrantz R, Olsson R, Danielsson A, et al.: A three year prospective study on serum tumor markers used for detecting cholangiocarcinoma in patients with primary sclerosing cholangitis. J Hepatol 1999, 30:669–673.
Petersen-Benz C, Stiehl A: Impact of dominant stenoses on the serum level of the tumour marker Ca 19-9 in patients with primary sclerosing cholangitis [in German]. Z Gastroenterol 2005, 43:587–590.
Burak K, Angulo P, Pasha TM, et al.: Incidence and risk factors for cholangiocarcinoma in primary sclerosing cholangitis. Am J Gastroenterol 2004, 99:523–526.
Broome U, Löfberg R, Veress B, Erikson LS: Primary sclerosing cholangitis and ulcerative colitis: evidence for increased neoplastic potential. Hepatology 1995, 22:1404–1408.
Uehara T, Hamano H, Kawa S, et al.: Distinct clinico-pathological entity ‘autoimmune pancreatitis-associated sclerosing cholangitis’. Pathol Int 2005, 55:405–411.
Engler S, Elsing C, Flechtenmacher C, et al.: Progressive sclerosing cholangitis after septic shock: a new variant of vanishing bile duct disorder. Gut 2003, 52:688–689.
Stiehl A, Walker S, Stiehl L, et al.: Effects of ursodeoxycholic acid on liver and bile duct disease in primary sclerosing cholangitis. A 3 year pilot study with a placebo-controlled study period. J Hepatol 1994, 20:57–64.
Beuers U, Spengler U, Kruis W, et al.: Ursodeoxycholic acid for treatment of primary sclerosing cholangitis: a placebo controlled trial. Hepatology 1992, 16:707–714.
Lindor KD: Ursodiol for primary sclerosing cholangitis. Mayo Primary Sclerosing Cholangitis-Ursodeoxycholic Acid Study Group. N Engl J Med 1997, 336:691–695.
Mitchell SA, Bansi D, Hunt N, et al.: A preliminary trial of high dose ursodeoxycholic acid in primary sclerosing cholangitis. Gastroenterology 2001, 121:900–907.
Harnois DM, Angulo P, Jorgensen RA, et al.: High-dose ursodeoxycholic acid as therapy for patients with primary sclerosing cholangitis. Am J Gastroenterol 2001, 96:1558–1562.
Olsson R, Boberg KM, Schaffalisky de Muckadell O, et al.: High-dose ursodeoxycholic acid in primary sclerosing cholangitis: a 5-year multicenter, randomized, controlled study. Gastroenterology 2005, 129:1464–1472.
Sauer P, Benz C, Rudolph G, et al.: Influence of cholestasis on absorption of ursodeoxycholic acid. Dig Dis Sci 1999, 44:817–822.
Rost D, Rudolph G, Kloeters-Plachky P, Stiehl A: Effect of high-dose ursodeoxycholic acid on its biliary enrichment in primary sclerosing cholangitis. Hepatology 2004, 40:693–698.
Rost D, Rudolph G, Kloeters-Plachky P, Stiehl A: Effect of colitis and ileoanal pouch on biliary enrichment of ursodeoxycholic acid in primary sclerosing cholangitis. Dig Dis Sci 2006, 51:618–622.
Cullen SN, Chapman RW: The medical management of primary sclerosing cholangitis. Semin Liver Dis 2006, 26:52–61.
Earnest DL, Holubec H, Wali RK, et al.: Chemoprevention of azomethane-induced colonic carcinogenesis by supplemental dietary ursodeoxycholic acid. Cancer Res 1994, 54:5071–5074.
Stiehl A, Raedsch R, Rudolph G: Ileal excretion of bile acids: comparison with biliary bile composition and effect of ursodeoxycholic acid treatment. Gastroenterology 1988, 94:1201–1206.
Tung BY, Emond MJ, Haggitt RC, et al.: Ursodiol use is associated with lower prevalence of colonic neoplasia in patients with ulcerative colitis and primary sclerosing cholangitis. Ann Intern Med 2001, 134:89–95.
Pardi DS, Loftus EV, Kremers WK, et al.: Ursodeoxycholic acid as a chemoprotective agent in patients with ulcerative colitis and primary sclerosing cholangitis. Gastroenterology 2003, 124:889–893.
Wolf JM, Rybicki LA, Lashner BA: The impact of ursodeoxycholic acid on cancer, dysplasia and mortality in ulcerative colitis patients with primary sclerosing cholangitis. Aliment Pharmacol Ther 2005, 22:783–788.
Stiehl A, Rudolph G, Kloeters-Plachky P, et al.: Development of bile duct stenoses in patients with primary sclerosing cholangitis treated with ursodeoxycholic acid: outcome after endoscopic treatment. J Hepatol 2002, 36:151–156.
Brandsaeter B, Isoniemi H, Broome U, et al.: Liver transplantation for primary sclerosing cholangitis; predictors and consequences of hepatobiliary malignancy. J Hepatol 2004, 40:815–822.
Boberg KM, Egeland T, Schrumpf E: Long-term effect of corticoid treatment in primary sclerosing cholangitis patients. Scand J Gastroenterol 2003, 38:991–995.
Olsson R, Björnsson E, Bäckman L, et al.: Bile duct bacterial isolates in primary sclerosing cholangitis: a study of explanted livers. J Hepatol 1998, 28:426–432.
Färkkilä M, Karvonen AL, Nurmi H, et al.: Metronidazole and ursodeoxycholic acid for primary sclerosing cholangitis: a randomized placebo-controlled trial. Hepatology 2004, 40:1379–1386.
Kulaksiz H, Rudolph G, Kloeters-Plachky P, et al.: Biliary candida infections in primary sclerosing cholangitis. J Hepatol 2006, 45:711–716.
Ghent CN, Carruthers SG: Treatment of pruritus in primary biliary cirrhosis with rifampin. Results of a double-blind, crossover, randomized trial. Gastroenterology 1988, 94:488–493.
Podesta A, Lopez P, Terg R, et al.: Treatment of pruritus of primary biliary cirrhosis with rifampin. Dig Dis Sci 1991, 36:216–220.
Bachs L, Pares A, Elena M, et al.: Effects of long-term rifampicin administration in primary biliary cirrhosis. Gastroenterology 1992, 102:2077–2080.
Bergasa NV, Talbot TL, Alling DW, et al.: A controlled trial of naloxone infusions for the prurittus of chronic cholestasis. Gastroenterology 1992, 102:544–549.
Bergasa NV, Schmitt JM, Talbot TL, et al.: Open-label trial of oral nalmefene therapy for pruritus of cholestasis. Hepatology 1998, 27:679–684.
Wolfhagen FH, Sternieri E, Hop WC, et al.: Oral naltrexone treatment for cholestatic pruritus: a double-blind, placebo-controlled study. Gastroenterology 1997, 113:1264–1269.
Muller C, Pongratz S, Pidlich J, et al.: Treatment of pruritus in chronic liver disease with 5-hydroxytryptamine receptor type 3 antagonist ondansetron: a randomized, placebo-controlled, double-blind cross-over trial. Eur J Gastroenterol Hepatol 1998, 10:865–870.
Grijm R, Huibregtse K, Bartelsman J, et al.: Therapeutic investigations in primary sclerosing cholangitis. Dig Dis Sci 1986, 31:792–798.
Johnson GK, Geenen JE, Venu RP, Hogan WJ: Endoscopic treatment of biliary duct strictures in sclerosing cholangitis: follow-up assessment of a new therapeutic approach. Gastrointest Endosc 1987, 33:9–12.
Lee JG, Schutz SM, England RE, et al.: Endoscopic therapy of sclerosing cholangitis. Hepatology 1995, 21:661–667.
Van Milligen de Wit AW, van Bracht J, Rauws EA, et al.: Endoscopic stent therapy for dominant extrahepatic bile duct strictures in primary sclerosing cholangitis. Gastrointest Endosc 1996, 44:293–299.
Stiehl A, Rudolph G, Sauer P, et al.: Efficacy of ursodeoxycholic acid and endoscopic dilation of major duct stenoses in primary sclerosing cholangitis. An 8-year prospective study. J Hepatol 1997, 26:560–566.
Ponsioen CY, Lam K, van Milligen de Wit AW, et al.: Four years experience with short-term stenting in primary sclerosing cholangitis. Am J Gastroenterol 1999, 94:2403–2407.
Kaya M, Petersen BT, Angulo P, et al.: Balloon dilatation compared to stenting of dominant strictures in primary sclerosing cholangitis. Am J Gastroenterol 2001, 96:1059–1066.
Baluyut AR, Sherman S, Lehman GA, et al.: Impact of endoscopic therapy on the survival of patients with primary sclerosing cholangitis. Gastrointest Endosc 2001, 53:308–312.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Rost, D., Kulaksiz, H. & Stiehl, A. Treatment of primary sclerosing cholangitis. Curr Treat Options Gastro 10, 111–119 (2007). https://doi.org/10.1007/s11938-007-0063-6
Published:
Issue Date:
DOI: https://doi.org/10.1007/s11938-007-0063-6