Tofacitinib in Pediatric Ulcerative Colitis: A Retrospective Multicenter Experience.

Autor:	Ledder O; Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel.; Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel., Dolinger M; Division of Pediatric Gastroenterology, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Dubinsky MC; Division of Pediatric Gastroenterology, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Stein R; Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, PA, USA., Vellanki S; Division of Pediatric Gastroenterology, Hepatology and Nutrition, UT Southwestern Medical Center and Children's Medical Center, Dallas, TX, USA., Buckuk R; Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel., Fatima A; Pediatric IBD Center, Beaumont Children's Hospital, Royal Oak, MI, USA., Suskind DL; Division of Gastroenterology, Seattle Children's Hospital Inflammatory Bowel Disease Center, Seattle, WA, USA., Scarlett J; Division of Gastroenterology, Seattle Children's Hospital Inflammatory Bowel Disease Center, Seattle, WA, USA., Röser D; Pediatric Department, Copenhagen University Hospital, Hvidovre, Denmark., Shouval DS; Institute of Gastroenterology Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petach Tikva.; Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel., Meyer G; Division of Pediatric Gastroenterology, Nemours Children's Hospital, Wilmington, DE, USA., Rios ZM; Division of Pediatric Gastroenterology, Nemours Children's Hospital, Wilmington, DE, USA., Pujol-Muncunill G; Pediatric Gastroenterology, Hepatology and Nutrition Department, Hospital Sant Joan de Deu, Barcelona, Spain., Lozano A; Pediatric Gastroenterology, Hepatology and Nutrition Department, Hospital Sant Joan de Deu, Barcelona, Spain., Kolho KL; Pediatric Gastroenterology Department, Children's Hospital HUS, Helsinki University, Helsinki, Finland., Rohani P; Pediatric Gastroenterology and Hepatology Research Center, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran., Hussey S; Department of Paediatric Gastroenterology, Children's Health Ireland, Royal College of Surgeons of Ireland and University College Dublin, Dublin, Ireland., de Mejj T; Department of Pediatric Gastroenterology, AG&M Research Institute, Amsterdam UMC, Amsterdam, the Netherlands., Ayers T; Division of Pediatric Gastroenterology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA., Navas-López VM; Pediatric Gastroenterology and Nutrition Unit, Instituto de Investigación Biomédica de Málaga, Hospital Regional Universitario de Málaga, Málaga, Spain., Turner D; Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel.; Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel., Tzivinikos C; Paediatric Gastroenterology Department, Al Jalila Children's Specialty Hospital, Mohammed Bin Rashid University, Dubai, United Arab Emirates.
Jazyk:	angličtina
Zdroj:	Inflammatory bowel diseases [Inflamm Bowel Dis] 2024 Jun 03. Date of Electronic Publication: 2024 Jun 03.
DOI:	10.1093/ibd/izae112
Abstrakt:	Background: Tofacitinib has recently been approved for treatment of moderate-to-severe ulcerative colitis (UC) in adults, yet pediatric data are limited. This international multicenter study describes the effectiveness and safety of tofacitinib in pediatric UC. Methods: This is a retrospective review of children diagnosed with UC treated with tofacitinib from 16 pediatric centers internationally. The primary outcome was week 8 corticosteroid-free clinical remission (Pediatric Ulcerative Colitis Activity Index <10). Secondary outcomes were clinical response (≥20-point decrease in Pediatric Ulcerative Colitis Activity Index) at week 8, corticosteroid-free clinical remission at week 24, and colectomy rate and adverse safety events through to last follow-up. The primary outcome was calculated by the intention-to-treat principle. Results: We included 101 children with a mean age at diagnosis of 12.8 ± 2.8 years and a median disease duration of 20 months (interquartile range [IQR], 10-39 months). All had treatment failure with at least 1 biologic agent, and 36 (36%) had treatment failure with 3 agents. Median follow-up was 24 weeks (IQR, 16-54 weeks). Sixteen (16%) children achieved corticosteroid-free clinical remission at week 8, and an additional 30 (30%) demonstrated clinical response. Twenty (23%) of 88 children achieved corticosteroid-free clinical remission at week 24. A total of 25 (25%) children underwent colectomy by median 86 days (IQR, 36-130 days). No serious drug-related adverse events were reported; there was 1 case of herpes zoster and 2 cases of minor blood test perturbations. Conclusions: In this largest real-life pediatric cohort to date, tofacitinib was effective in at least 16% of patients with highly refractory UC by week 8. Adverse events were minor and largely consistent with adult data. (© The Author(s) 2024. Published by Oxford University Press on behalf of Crohn’s & Colitis Foundation. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.)
Databáze:	MEDLINE
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