Efficacy of Oral Vancomycin in Treating Primary Sclerosing Cholangitis in Pediatric Patients

Although there is no approved treatment available for primary sclerosing cholangitis (PSC), in the pediatric population, off-label use of ursodeoxycholic acid (UDCA), oral vancomycin (specifically thought to treat gut dysbiosis in PSC), or both is common. It is unknown whether these therapies affect long-term outcome.

This retrospective study involved patients from the international Pediatric PSC Consortium registry in which 85% of PSC patients received UDCA, vancomycin, or both. Outcomes were determined in patients receiving UDCA, vancomycin, or observation only, who were propensity-score–matched in a 1:1:1 ratio.

The three 88-member groups had similar demographics, baseline biochemistry, and hepatic fibrosis. Over a 1-year period, intention…