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.

1st Pediatric Clinic University of Athens Athens Greece

Department of Gastroenterology Hepatology and Nutrition Children's National Medical Center Washington DC

Department of Pediatrics and Child Health University of Manitoba Winnipeg Manitoba Canada

Department of Pediatrics and Pediatric Neuropsychiatry Sapienza University of Rome Rome Italy

Department of Pediatrics Cincinnati Children's Hospital Medical Center Cincinnati OH

Department of Pediatrics Emory University School of Medicine Atlanta GA

Department of Pediatrics Harvard University Boston MA

Department of Pediatrics Medical College of Wisconsin Milwaukee WI

Department of Pediatrics Oklahoma University Oklahoma City OK

Department of Pediatrics Palacky University Olomouc Czech Republic

Department of Pediatrics Texas Children's Hospital Houston TX

Department of Pediatrics The Children's Hospital of Philadelphia Philadelphia PA

Department of Pediatrics The Hospital for Sick Children University of Toronto Toronto Ontario Canada

Department of Pediatrics University of California San Francisco San Francisco CA

Department of Pediatrics University of Colorado School of Medicine Aurora CO

Department of Pediatrics University of Helsinki Helsinki Finland

Department of Pediatrics University of Naples Federico 2 Naples Italy

Department of Pediatrics University of Pittsburgh Medical Center Pittsburgh PA

Department of Pediatrics University of Rochester Medical Center Rochester NY

Department of Pediatrics University of Utah Salt Lake City UT

Department of Pediatrics Yale University School of Medicine New Haven CT

Division of Gastroenterology Liver and Nutrition The Dana Dwek Children's Hospital Tel Aviv University Tel Aviv Israel

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