Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis.
Mark R DeneauCara MackDouglas MogulEmily R PeritoPamela L ValentinoAchiya Z AmirMatthew DiGuglielmoLaura G DraijerWael El-MataryKatryn N FuruyaNitika GuptaJessica T HochbergSimon HorslenM Kyle JensenMaureen M JonasNanda KerkarBart G P KootTrevor J LabordaChristine K LeeKathleen M LoomesMercedes MartinezAlexander MiethkeTamir MilohSaeed MohammedNadia OvchinskyGirish RaoAmanda RicciutoPushpa SathyaKathleen B SchwarzUzma ShahRuchi SinghBernadette VitolaAndréanne N ZizzoStephen L GutheryPublished in: Hepatology (Baltimore, Md.) (2021)
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.