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

Trevor J Laborda1, Amanda Ricciuto2, Madeleine Aumar3

  • 1University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT.

Insights

Vedolizumab (VDZ) did not improve liver outcomes in pediatric patients with primary sclerosing cholangitis and inflammatory bowel disease (PSC-IBD). Liver disease worsened in those with medically refractory IBD, suggesting progressive liver damage.

Area of Science:

  • Gastroenterology
  • Hepatology
  • Immunology

Background:

  • Primary sclerosing cholangitis (PSC) frequently co-occurs with inflammatory bowel disease (IBD).
  • Vedolizumab (VDZ), an anti-α4β7 integrin antibody, targets lymphocyte homing to the gut and liver via MAdCAM-1.
  • Investigating VDZ's efficacy in pediatric PSC-IBD patients is crucial due to shared pathophysiology.

Purpose of the Study:

  • To evaluate liver outcomes in pediatric patients with PSC-IBD treated with VDZ.
  • To assess the impact of VDZ on liver biochemistry and IBD activity in this cohort.
  • To identify factors associated with liver response to VDZ therapy.

Main Methods:

  • Retrospective analysis of 37 pediatric patients with PSC-IBD from the Pediatric PSC Consortium.
  • Collection of demographic, clinical, biochemical, radiological, and histopathological data up to 1 year of VDZ therapy.
  • Definition of liver biochemical response as a ≥75% reduction in GGT or GGT <50 IU/L; IBD response as improved endoscopic/activity scores.

Main Results:

  • 22% of patients with abnormal baseline GGT achieved liver biochemical response.
  • 32% achieved IBD remission, 30% clinical response, and 38% no response.
  • Liver biochemistry worsened in VDZ-unresponsive IBD patients but remained stable in those in remission (P=0.066).

Conclusions:

  • Vedolizumab did not demonstrate significant improvement in liver biochemistry for pediatric PSC-IBD patients.
  • Progressive liver disease may be more prevalent in patients with medically refractory IBD.
  • VDZ's efficacy in improving liver outcomes in pediatric PSC-IBD warrants further investigation.
Abstract

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