@article{3105992,
    title = "Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course",
    author = "Deneau, M. and Perito, E. and Ricciuto, A. and Gupta, N. and Kamath, B.M. and Palle, S. and Vitola, B. and Smolka, V. and Ferrari, F. and Amir, A.Z. and Miloh, T. and Papadopoulou, A. and Mohan, P. and Mack, C. and Kolho, K.-L. and Iorio, R. and El-Matary, W. and Venkat, V. and Chan, A. and Saubermann, L. and Valentino, P.L. and Shah, U. and Miethke, A. and Lin, H. and Jensen, M.K.",
    journal = "Journal of Pediatric Neurosciences",
    year = "2019",
    volume = "209",
    pages = "92-96.e1",
    publisher = "Mosby Year Book Inc",
    issn = "1817-1745",
    doi = "10.1016/j.jpeds.2019.01.039",
    keywords = "alanine aminotransferase;  alkaline phosphatase;  aspartate aminotransferase;  bilirubin;  gamma glutamyltransferase;  serum albumin;  ursodeoxycholic acid;  biological marker;  gamma glutamyltransferase;  ursodeoxycholic acid, adolescent;  albumin blood level;  Article;  child;  childhood mortality;  clinical outcome;  Crohn disease;  disease course;  event free survival;  female;  gamma glutamyl transferase blood level;  human;  laboratory test;  liver fibrosis;  liver transplantation;  long term survival;  major clinical study;  male;  medical record review;  phenotype;  platelet count;  portal hypertension;  predictive value;  primary sclerosing cholangitis;  priority journal;  retrospective study;  risk reduction;  survival time;  treatment response;  analysis of variance;  blood;  clinical trial;  cohort analysis;  comparative study;  follow up;  liver function test;  multicenter study;  predictive value;  sclerosing cholangitis;  severity of illness index;  time factor;  treatment failure;  treatment outcome, Adolescent;  Analysis of Variance;  Biomarkers;  Child;  Cholangitis, Sclerosing;  Cohort Studies;  Female;  Follow-Up Studies;  gamma-Glutamyltransferase;  Humans;  Liver Function Tests;  Male;  Predictive Value of Tests;  Retrospective Studies;  Severity of Illness Index;  Time Factors;  Treatment Failure;  Treatment Outcome;  Ursodeoxycholic Acid",
    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. © 2019 Elsevier Inc."
}