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Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course
M. Deneau, E. Perito, A. Ricciuto, N. Gupta, BM. Kamath, S. Palle, B. Vitola, V. Smolka, F. Ferrari, AZ. Amir, T. Miloh, A. Papadopoulou, P. Mohan, C. Mack, KL. Kolho, R. Iorio, W. El-Matary, V. Venkat, A. Chan, L. Saubermann, PL. Valentino, U....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu srovnávací studie, časopisecké články, multicentrická studie, Research Support, N.I.H., Extramural, práce podpořená grantem
Grantová podpora
UL1 RR025764
NCRR NIH HHS - United States
UL1 TR001863
NCATS NIH HHS - United States
P30 DK078392
NIDDK NIH HHS - United States
UL1 TR000105
NCATS NIH HHS - United States
UL1 TR002538
NCATS NIH HHS - United States
KL2 TR001065
NCATS NIH HHS - United States
- MeSH
- analýza rozptylu MeSH
- biologické markery krev MeSH
- časové faktory MeSH
- dítě MeSH
- gama-glutamyltransferasa krev MeSH
- jaterní testy MeSH
- kohortové studie MeSH
- kyselina ursodeoxycholová terapeutické užití MeSH
- lidé MeSH
- mladiství MeSH
- následné studie MeSH
- neúspěšná terapie MeSH
- prediktivní hodnota testů MeSH
- retrospektivní studie MeSH
- sklerozující cholangitida krev farmakoterapie MeSH
- stupeň závažnosti nemoci MeSH
- výsledek terapie MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- Research Support, N.I.H., Extramural MeSH
- srovnávací studie MeSH
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 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
Citace poskytuje Crossref.org
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- $a Deneau, Mark $u Department of Pediatrics, University of Utah, Salt Lake City, UT. Electronic address: mark.deneau@hsc.utah.edu.
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