Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis
dc.contributor.author | Deneau, Mark R. | |
dc.contributor.author | Mack, Cara | |
dc.contributor.author | Mogul, Douglas | |
dc.contributor.author | Perito, Emily R. | |
dc.contributor.author | Valentino, Pamela L. | |
dc.contributor.author | Amir, Achiya Z. | |
dc.contributor.author | DiGuglielmo, Matthew | |
dc.contributor.author | Draijer, Laura G. | |
dc.contributor.author | El-Matary, Wael | |
dc.contributor.author | Furuya, Katryn N. | |
dc.contributor.author | Gupta, Nitika | |
dc.contributor.author | Hochberg, Jessica T. | |
dc.contributor.author | Horslen, Simon | |
dc.contributor.author | Jensen, M. Kyle | |
dc.contributor.author | Jonas, Maureen M. | |
dc.contributor.author | Kerkar, Nanda | |
dc.contributor.author | Koot, Bart G. P. | |
dc.contributor.author | Laborda, Trevor J. | |
dc.contributor.author | Lee, Christine K. | |
dc.contributor.author | Loomes, Kathleen M. | |
dc.contributor.author | Martinez, Mercedes | |
dc.contributor.author | Miethke, Alexander | |
dc.contributor.author | Miloh, Tamir | |
dc.contributor.author | Mohammad, Saeed | |
dc.contributor.author | Ovchinsky, Nadia | |
dc.contributor.author | Rao, Girish | |
dc.contributor.author | Ricciuto, Amanda | |
dc.contributor.author | Sathya, Pushpa | |
dc.contributor.author | Schwarz, Kathleen B. | |
dc.contributor.author | Shah, Uzma | |
dc.contributor.author | Singh, Ruchi | |
dc.contributor.author | Vitola, Bernadette | |
dc.contributor.author | Zizzo, Andréanne | |
dc.contributor.author | Guthery, Stephen L. | |
dc.contributor.department | Pediatrics, School of Medicine | |
dc.date.accessioned | 2024-03-20T10:35:48Z | |
dc.date.available | 2024-03-20T10:35:48Z | |
dc.date.issued | 2021 | |
dc.description.abstract | 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. | |
dc.eprint.version | Author's manuscript | |
dc.identifier.citation | Deneau MR, Mack C, Mogul D, et al. Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis. Hepatology. 2021;73(3):1061-1073. doi:10.1002/hep.31560 | |
dc.identifier.uri | https://hdl.handle.net/1805/39358 | |
dc.language.iso | en_US | |
dc.publisher | Wolters Kluwer | |
dc.relation.isversionof | 10.1002/hep.31560 | |
dc.relation.journal | Hepatology | |
dc.rights | Publisher Policy | |
dc.source | PMC | |
dc.subject | Bilirubin | |
dc.subject | Serum albumin | |
dc.subject | Ursodeoxycholic acid | |
dc.subject | Vancomycin | |
dc.title | Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis | |
dc.type | Article |