Vedolizumab Therapy in Children With Primary Sclerosing Cholangitis: Data From the Pediatric Primary Sclerosing Cholangitis Consortium

Trevor J. Laborda, Amanda Ricciuto, Madeleine Aumar, Nicholas Carman, Matthew DiGuglielmo, Laura G. Draijer, Katryn N. Furuya, Nitika Gupta, Bart G.P. Koot, Kathleen M. Loomes, Ellina Lytvyak, Mercedes Martinez, Tamir Miloh, Aldo J. Montano-Loza, Emily R. Perito, Pushpa Sathya, Uzma Shah, Eyal Shteyer, Ruchi Singh, Amy TaylorPamela L. Valentino, Bernadette Vitola, Melissa Zerofsky, Andréanne Zizzo, Mark R. Deneau

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


OBJECTIVES: Most patients with primary sclerosing cholangitis (PSC) also have inflammatory bowel disease (IBD). The liver and colon express MAdCAM-1, a target of lymphocyte homing integrins. Vedolizumab (VDZ) is an α4β7 integrin antibody used to treat IBD. We investigated liver outcomes in children with PSC-IBD treated with VDZ. METHODS: Patients were identified within the Pediatric PSC Consortium, a multicenter research registry. Retrospective demographic, phenotypic, biochemical, radiological, histopathologic and IBD data for up to 1 year of VDZ therapy were collected. Liver biochemical and IBD responses were defined as: a 75% or greater reduction in initial γ-glutamyltransferase (GGT), or a GGT that fell to <50 IU/L and improved Mayo endoscopy grade or IBD activity scores after 9 to 12 months. RESULTS: Thirty-seven patients were identified from 19 centers. VDZ was initiated at median age of 16 years [IQR 15-18], 69% were male, 65% had large duct involvement, 19% had (Metavir F3/F4) fibrosis and 59% had ulcerative colitis. Of 32 patients with abnormal GGT at baseline, 22% had a liver biochemical response after 9 to 12 months. For IBD, 32% achieved remission, 30% had a clinical response, and 38% had no response. Final GGT after 9 to 12 months was 51 [IQR 28-71] in IBD patients in remission versus 127 [IQR 63-226] in those with active IBD, (P = 0.066). CONCLUSIONS: Liver biochemistry worsened over time in IBD unresponsive to VDZ but remained unchanged in IBD patients in remission. VDZ did not improve liver biochemistry in pediatric PSC-IBD. Progressive liver disease may be more common in patients with medically refractory IBD.

Original languageEnglish (US)
Pages (from-to)459-464
Number of pages6
JournalJournal of pediatric gastroenterology and nutrition
Issue number4
StatePublished - Oct 1 2020

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Gastroenterology


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