BOSTON, MA—“Real world use of ursodeoxycholic acid in a large cohort of children with primary sclerosing cholangitis did not improve liver outcomes,” a retrospective study presented at The Liver Meeting® 2016 concluded. 

“Many providers use ursodeoxycholic acid in children with primary sclerosing cholangitis despite a lack of convincing evidence of efficacy, and potential harm seen in adults,” noted Mark R. Deneau, MD, Pediatric Gastroenterology, University of Utah, Salt Lake City, UT. 

Using data from the Pediatric Primary Sclerosing Cholangitis Consortium, a collaboration among 26 nations and clinical liver disease centers, Dr. Deneau and colleagues evaluated the effectiveness of routine use of ursodeoxycholic acid. 

They performed a survival analysis of 500 children in the cohort, from diagnosis of primary sclerosing cholangitis to diagnosis of portal hypertensive complications (ascites, encephalopathy, or esophageal varices); biliary complication (stricture requiring dilation, stent placement, or drainage); liver transplantation; cholangiocarcinoma; or liver-related death. A composite outcome was termed “complicated liver disease.” 

A total of 34 patients were excluded from the analysis because they had a complicated liver disease outcome at or within two months of a diagnosis of primary sclerosing cholangitis. 

“Of the remaining patients, 78% were treated chronically with ursodeoxycholic acid,” the study authors noted. 

The mean daily reported dose was 14.5mg/kg/day (range, 10–30mg); however, the dose was recorded for only 20% of the patients. 

No evidence of a bias towards ursodeoxycholic acid treatment in patients with more severe disease was found. Similar levels of baseline GGT (274 vs. 273 U/L; P=0.949), total bilirubin (1.4 vs. 1.6mg/dL; P=0.563), and MELD score (-1 vs. 0; P=0.282), were observed in patients who were untreated and treated, respectively. 

For patients treated with ursodeoxycholic acid compared with those who were not, the observed 5-year survival time was worse in every outcome category. These were progression to portal hypertension (P=0.238), transplant-free survival after diagnosis of portal hypertension (P=0.340), progression to biliary complications (P=0.158), transplant-free survival after diagnosis of biliary complication (P=0.992), progression to complicated liver disease (P=0.633), or survival with native liver (P=0.286). 

These results remained unchanged when subgroups were analyzed separately: primary sclerosing cholangitis plus autoimmune hepatitis overlap, large or small duct disease, primary sclerosing cholangitis plus inflammatory bowel disease, and primary sclerosing cholangitis without inflammatory bowel disease. 

“It is unclear from these data if certain doses of ursodeoxycholic acid in certain patient subgroups may have been beneficial, but broad use of ursodeoxycholic acid in children with primary sclerosing cholangitis did not prevent adverse liver outcomes,” the study authors concluded.