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

Autor: Laborda TJ; University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT., Ricciuto A; University of Toronto, Toronto, Ontario, Canada., Aumar M; University of Lille, CHU Lille, Lille, France., Carman N; Children's Hospital of Eastern Ontario, Ottawa, ON., DiGuglielmo M; Nemours Alfred I duPont Hospital for Children, Wilmington, DE., Draijer LG; Amsterdam University Medical Center, The Netherlands., Furuya KN; University of Wisconsin at Madison School of Medicine and Public Health, Madison, WI., Gupta N; Emory University School of Medicine, Atlanta, GA., Koot BGP; Amsterdam University Medical Center, The Netherlands., Loomes KM; Children's Hospital of Philadelphia, Philadelphia, PA., Lytvyak E; University of Alberta, Edmonton, Alberta, Canada., Martinez M; Columbia University, New York, NY., Miloh T; University of Miami, Miami, Florida., Montano-Loza AJ; University of Alberta, Edmonton, Alberta, Canada., Perito ER; University of California, San Francisco, CA., Sathya P; Memorial University, St. John's, Newfoundland and Labrador, Canada., Shah U; Massachusetts General Hospital, Harvard Medical School, Boston, MA., Shteyer E; The Juliet Keidan Institute of Pediatric gastroenterology and Nutrition, Shaare Zedek Medical Center, Jerusalem, Israel., Singh R; Cincinnati Children's Hospital Medical Center, Cincinnati, OH., Taylor A; Cincinnati Children's Hospital Medical Center, Cincinnati, OH., Valentino PL; Yale University School of Medicine, New Haven, CT., Vitola B; Medical College of Wisconsin, Milwaukee, WI., Zerofsky M; University of California San Francisco, San Francisco, CA., Zizzo A; London Health Sciences Center, Western University, London, Ontario, Canada., Deneau MR; University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT.
Jazyk: angličtina
Zdroj: Journal of pediatric gastroenterology and nutrition [J Pediatr Gastroenterol Nutr] 2020 Oct; Vol. 71 (4), pp. 459-464.
DOI: 10.1097/MPG.0000000000002855
Abstrakt: 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.
Databáze: MEDLINE
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