Aug. 31, 2009 A national team of researchers led by scientists at Mayo Clinic has found that a common treatment for primary sclerosing cholangitis, a chronic liver disease, is not helpful for patients, according to a study published this month in the journal Hepatology.
Primary sclerosing cholangitis (PSC) is a disease of the bile ducts. In this case, the term "cholangitis" refers to inflammation of the bile ducts, while "sclerosing" describes the hardening and scarring of the bile ducts that result from chronic inflammation.
"Primary sclerosing cholangitis is a serious liver disease lacking an effective medical therapy," says Keith Lindor, M.D., Mayo Clinic gastroenterologist and the study's lead researcher. "Some studies have shown that the use of ursodeoxycholic acid, a naturally occurring bile acid, may be a potential solution for patients. Our research, however, showed long-term use of this treatment in high dosages is not suitable for patients."
In this six-year, multicenter trial, 150 patients were enrolled in the study to determine the effectiveness of ursodeoxycholic acid (UDCA) in treatment of PSC. Seventy-six patients were treated with higher doses (28 to 30 mg/kg/day) of UDCA and 74 patients were given a placebo. Serious adverse events were more common in the UDCA group than the placebo group, which prompted researchers to halt the study. UDCA has been thought to be a possible treatment solution for PSC patients, but this trial indicates that the drug, used at this higher dose, is not helpful.
"All of us were surprised that the higher doses of UDCA did not help; in fact, the risk of developing even more liver problems increased with the higher dosages," says Dr. Lindor. "While this was thought to be the best potential treatment for PSC, our study found that not to be the case."
Dr. Lindor says that patients who are currently on higher doses of UDCA should consult with their doctors. He also points out that these study findings highlight the need for more research to look into better treatment options for PSC.
PSC is a progressive disease that leads to liver damage and, eventually, liver failure. Liver transplant is the only known cure for PSC, but transplant is typically reserved for people with severe liver damage.
PSC most often affects people in their 30s to 50s. The average age at diagnosis is 40. However, the condition can arise in childhood. About 60 to 75 percent of people diagnosed with the disease are men. Approximately 70 percent of people with PSC have an associated disease such as inflammatory bowel disease, osteoporosis, gallbladder disease and bile duct cancer or cholangiocarcinoma. However, only 1 to 5 percent of people with inflammatory bowel disease have PSC.
Other members of the Mayo Clinic research team include M. Edwyn Harrison, M.D., Denise Harnois, D.O., Roberta Jorgensen, Jan Petz, Jill Keach, Julie Braaten, M.D., Ellen Miceli, Jeff Schmoll, Tanya Hoskin, Prabin Thapa and Felicity Enders, Ph.D. Other researchers include Kris Kowdley, M.D., and Jody Mooney, M.D., Virginia Mason Medical Center; Velimir Luketic, M.D., and Carol Sargeant, M.D., Virginia Commonwealth University School of Medicine; Timothy McCashland, M.D., and Tamara Bernard, M.D., University of Nebraska; and Alex Befeler, M.D., and Debra King, M.D., Saint Louis University.
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- Keith D. Lindor, Kris V. Kowdley, Velimir A. C. Luketic, M. Edwyn Harrison, Timothy McCashland, Alex S. Befeler, Denise Harnois, Roberta Jorgensen, Jan Petz, Jill Keach, Jody Mooney, Carol Sargeant, Julie Braaten, Tamara Bernard, Debra King, Ellen Miceli, Jeff Schmoll, Tanya Hoskin, Prabin Thapa, Felicity Enders. High Dose Ursodeoxycholic Acid for the Treatment of Primary Sclerosing Cholangitis. Hepatology, 2009 DOI: 10.1002/hep.23082
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