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ALEXANDRIA, VA – Data from a new study presented this week at The Liver Meeting Digital Experience® – held by the American Association for the Study of Liver Diseases – found that ursodeoxycholic acid (UDCA) treatment has significant, positive results for patients with primary sclerosing cholangitis (PSC), including reduced incidence of biliary tract cancer, reduced mortality and less need for liver transplant.

UDCA, a common cholesterol medication, can be used for the treatment of cholestatic liver diseases, however, a number of prospective clinical studies have had conflicting results and the therapy’s long-term impact on patients with PSC is unknown. To learn more about the possible links between UDCA, cancer and mortality risk, researchers in Japan conducted a study of 325 patients with primary sclerosing cholangitis who were part of a nationwide patient registry.

"Although a number of prospective clinical studies were conducted in the past to evaluate the efficacy of UDCA, results were conflicting mainly due to study design such as inappropriate definition of endpoints, limited number of patients and short follow-up," says the study’s co-author, Toshihiko Arizumi, MD, assistant professor in the Department of Internal Medicine at Teikyo University School of Medicine. "As a result, it remains unclear whether UDCA treatment really improves liver transplantation-free survival of PSC patients, and currently there is no effective treatment for PSC. On the other hand, a prospective, randomized, placebo-controlled, well-designed/powered study of UDCA is extremely unlikely to be conducted in the future, since this is an inexpensive, off-patent, old drug, and pharmaceutical companies do not have an interest in such a large-scale study. Therefore, we considered that this retrospective, well-characterized, cohort study would be the best option to demonstrate the clinical benefit of UDCA on long-term outcomes in patients with PSC. We don’t think that UDCA is associated with an increased risk of development of biliary tract cancer. Rather, biliary tract cancer is one of the most troublesome, even fatal, comorbidities of PSC itself. It would be fantastic if UDCA reduces a risk of this cancer in patients with PSC."

Patients in the study were registered via surveys from 2012 to 2015. Out of 435 patients with PSC, the researchers enrolled 325 who had no missing data on their: sex, age at diagnosis, blood test results (including platelet counts, albumin, bilirubin, aspartate aminotransferase, alanine aminotransferase and alkaline phosphatase), fibrous indexes at diagnosis, the presence of any symptom at diagnosis, any history or presence of inflammatory bowel disease, past treatment with UDCA and/or bezafibrate, any development of biliary tract (bile duct or gallbladder) cancer, and their long-term outcomes, such as whether they were alive or dead, or had undergone liver transplantation. The 325 patients included in the study included 187 males and 138 females, they and had a median age of 45.8. The median observational period for the study was 5.1 years. UDCA treatment was given to 278 patients, and bezafibrate treatment was given to 78 patients. During the observation period, 57 patients died, 24 patients had a liver transplant, and 26 patients developed biliary tract cancer, including 22 with bile duct cancer and four with gallbladder cancer.

The study’s findings show that UDCA treatment was associated with an improvement in liver transplantation-free survival and was likely to reduce incidence of biliary tract cancer in patients with PSC. Sensitivity analysis also indicated these similar, significant associations between UDCA treatment and positive outcomes. The researchers also conducted an inverse probability treatment weighting-adjusted model that indicated that UDCA is significantly associated with liver transplantation-free survival, but not with biliary tract cancer.

Based on the results of all of the analytical models used in the study, UDCA treatment is significantly associated with reduced mortality and the need for liver transplantation in patients with PSC, and is likely associated with a reduction in biliary tract cancer in this population.

"We believe these findings definitely provide the most robust evidence to show clinical benefit of UDCA in PSC for the time being, and this will encourage more treatment with UDCA in patients with PSC,” says Dr. Arizumi. “Nevertheless, we don’t consider that this cohort has enough statistical power due to its medium sample size. A large-scale cohort with an international collaboration is required to produce more convincing evidences of UDCA in PSC."

Dr. Arizumi will present these findings at The Liver Meeting Digital Experience™ during Parallel: Clinical Cholestatic (PBC/PSC) and Autoimmune Liver Diseases on November 15 at 5:30 PM ET. The corresponding abstract, "The Association of UDCA Treatment With Long-Term Outcome and Biliary Tract Cancer in Patients With Primary Sclerosing Cholangitis" can be found in the journal, HEPATOLOGY.

About the AASLD
AASLD is the leading organization of clinicians and researchers committed to preventing and curing liver disease. The work of our members has laid the foundation for the development of drugs used to treat patients with viral hepatitis. Access to care and support of liver disease research are at the center of AASLD’s advocacy efforts.