Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis.

Autor: Deneau MR; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Mack C; University of Colorado School of MedicineAuroraCO., Mogul D; Johns Hopkins UniversityBaltimoreMD., Perito ER; University of California, San FranciscoSan FranciscoCA., Valentino PL; Yale University School of MedicineNew HavenCT., Amir AZ; The Dana-Dwek Children's HospitalThe Tel-Aviv Medical CenterTel-Aviv UniversityTel AvivIsrael., DiGuglielmo M; Nemours/Alfred I. duPont Hospital for ChildrenWilmingtonDE., Draijer LG; Amsterdam University Medical CenterAmsterdamThe Netherlands., El-Matary W; University of ManitobaWinnipegManitobaCanada., Furuya KN; Mayo ClinicRochesterMN.; University of Wisconsin-Madison School of Medicine and Public HealthMadisonWI., Gupta N; Emory University School of MedicineAtlantaGA., Hochberg JT; University of MiamiMiamiFL., Horslen S; University of WashingtonSeattleWA., Jensen MK; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Jonas MM; Boston Children's Hospital and Harvard Medical SchoolBostonMA., Kerkar N; University of Rochester Medical CenterRochesterNY., Koot BGP; Amsterdam University Medical CenterAmsterdamThe Netherlands., Laborda TJ; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Lee CK; Boston Children's Hospital and Harvard Medical SchoolBostonMA., Loomes KM; Children's Hospital of PhiladelphiaPhiladelphiaPA., Martinez M; Columbia UniversityNew YorkNY., Miethke A; Cincinnati Children's Hospital Medical CenterCincinnatiOH., Miloh T; University of MiamiMiamiFL., Mohammad S; Lurie Children's HospitalChicagoIL., Ovchinsky N; Children's Hospital at MontefioreAlbert Einstein College of MedicineBronxNY., Rao G; Indiana UniversityIndianapolisIN., Ricciuto A; University of TorontoTorontoOntarioCanada., Sathya P; Memorial UniversitySt. John'sNewfoundland and LabradorCanada., Schwarz KB; Johns Hopkins UniversityBaltimoreMD.; University of California San DiegoSan DiegoCA., Shah U; Massachusetts General HospitalHarvard Medical SchoolBostonMA., Singh R; Cincinnati Children's Hospital Medical CenterCincinnatiOH., Vitola B; Medical College of WisconsinMilwaukeeWI., Zizzo A; London Health Sciences CenterWestern UniversityLondonOntarioCanada., Guthery SL; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT.
Jazyk: angličtina
Zdroj: Hepatology (Baltimore, Md.) [Hepatology] 2021 Mar; Vol. 73 (3), pp. 1061-1073.
DOI: 10.1002/hep.31560
Abstrakt: 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.
(© 2020 by the American Association for the Study of Liver Diseases.)
Databáze: MEDLINE
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