Faculty Collaboration Database

Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis. Hepatology 2021 Mar;73(3):1061-1073

Date

09/19/2020

Pubmed ID

32946600

Pubmed Central ID

PMC8557636

DOI

10.1002/hep.31560

Scopus ID

2-s2.0-85096470448 (requires institutional sign-in at Scopus site)   50 Citations

Abstract

BACKGROUND AND AIMS: Many children with primary sclerosing cholangitis (PSC) receive oral vancomycin therapy (OVT) or ursodeoxycholic acid (UDCA). There is a paucity of data on whether these medications improve outcomes.

APPROACH AND RESULTS: We analyzed retrospective data from the Pediatric PSC Consortium. Children treated with OVT were matched 1:1:1 to those treated with UDCA or managed with observation (no treatment) based on the closest propensity score, ensuring similar baseline characteristics. Two hundred sixty-four patients (88 each with OVT, UDCA, or observation) had matching propensity scores and were similar in demographics, phenotype, immunosuppression, baseline biochemistry, and hepatic fibrosis. After 1 year in an intention-to-treat analysis, all outcome metrics were similar regardless of treatment group. In OVT, UDCA, and untreated groups, respectively: Gamma-glutamyltransferase normalized in 53%, 49%, and 52% (P = not significant [NS]), liver fibrosis stage was improved in 20%, 13%, and 18% and worsened in 11%, 29%, and 18% (P = NS), and the 5-year probability of liver transplant listing was 21%, 10%, and 12% (P = NS). Favorable outcome was associated with having a mild phenotype of PSC and minimal hepatic fibrosis.

CONCLUSIONS: We presented the largest-ever description of outcomes on OVT in PSC and compared them to carefully matched patients on UDCA or no therapy. Neither OVT nor UDCA showed improvement in outcomes compared to a strategy of observation. Patients progressed to end-stage liver disease at similar rates. Spontaneous normalization of biochemistry is common in children receiving no therapy, particularly in the majority of children with a mild phenotype and an early stage of disease. Placebo-controlled treatment trials are needed to identify effective treatments for pediatric PSC.

Author List

Deneau MR, Mack C, Mogul D, Perito ER, Valentino PL, Amir AZ, DiGuglielmo M, Draijer LG, El-Matary W, Furuya KN, Gupta N, Hochberg JT, Horslen S, Jensen MK, Jonas MM, Kerkar N, Koot BGP, Laborda TJ, Lee CK, Loomes KM, Martinez M, Miethke A, Miloh T, Mohammad S, Ovchinsky N, Rao G, Ricciuto A, Sathya P, Schwarz KB, Shah U, Singh R, Vitola B, Zizzo A, Guthery SL

Authors

Cara Lynn Mack MD Chief, Professor in the Pediatrics department at Medical College of Wisconsin
Bernadette Vitola MD, MPH Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Administration, Oral
Adolescent
Bilirubin
Child
Cholangitis, Sclerosing
Female
Humans
Male
Propensity Score
Retrospective Studies
Serum Albumin
Treatment Outcome
Ursodeoxycholic Acid
Vancomycin