Faculty Collaboration Database

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course. J Pediatr 2019 Jun;209:92-96.e1

Date

03/18/2019

Pubmed ID

30878206

Pubmed Central ID

PMC6535363

DOI

10.1016/j.jpeds.2019.01.039

Scopus ID

2-s2.0-85062695603 (requires institutional sign-in at Scopus site)   17 Citations

Abstract

OBJECTIVE: To investigate patient factors predictive of gamma glutamyltransferase (GGT) normalization following ursodeoxycholic acid (UDCA) therapy in children with primary sclerosing cholangitis.

STUDY DESIGN: We retrospectively reviewed patient records at 46 centers. We included patients with a baseline serum GGT level ≥50 IU/L at diagnosis of primary sclerosing cholangitis who initiated UDCA therapy within 1 month and continued therapy for at least 1 year. We defined "normalization" as a GGT level <50 IU/L without experiencing portal hypertensive or dominant stricture events, liver transplantation, or death during the first year.

RESULTS: We identified 263 patients, median age 12.1 years at diagnosis, treated with UDCA at a median dose of 15 mg/kg/d. Normalization occurred in 46%. Patients with normalization had a lower prevalence of Crohn's disease, lower total bilirubin level, lower aspartate aminotransferase to platelet ratio index, greater platelet count, and greater serum albumin level at diagnosis. The 5-year survival with native liver was 99% in those patients who achieved normalization vs 77% in those who did not.

CONCLUSIONS: Less than one-half of the patients treated with UDCA have a complete GGT normalization in the first year after diagnosis, but this subset of patients has a favorable 5-year outcome. Normalization is less likely in patients with a Crohn's disease phenotype or a laboratory profile suggestive of more advanced hepatobiliary fibrosis. Patients who do not achieve normalization could reasonably stop UDCA, as they are likely not receiving clinical benefit. Alternative treatments with improved efficacy are needed, particularly for patients with already-advanced disease.

Author List

Deneau M, Perito E, Ricciuto A, Gupta N, Kamath BM, Palle S, Vitola B, Smolka V, Ferrari F, Amir AZ, Miloh T, Papadopoulou A, Mohan P, Mack C, Kolho KL, Iorio R, El-Matary W, Venkat V, Chan A, Saubermann L, Valentino PL, Shah U, Miethke A, Lin H, Jensen MK

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

Adolescent
Analysis of Variance
Biomarkers
Child
Cholangitis, Sclerosing
Cohort Studies
Female
Follow-Up Studies
Humans
Liver Function Tests
Male
Predictive Value of Tests
Retrospective Studies
Severity of Illness Index
Time Factors
Treatment Failure
Treatment Outcome
Ursodeoxycholic Acid
gamma-Glutamyltransferase