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Tofacitinib in Pediatric Ulcerative Colitis: A Retrospective Multicenter Experience Publisher Pubmed



Ledder O1, 2 ; Dolinger M3 ; Dubinsky MC3 ; Stein R4 ; Vellanki S5 ; Buckuk R1 ; Fatima A6 ; Suskind DL7 ; Scarlett J7 ; Roser D8 ; Shouval DS9, 10 ; Meyer G11 ; Rios ZM11 ; Pujolmuncunill G12 Show All Authors
Authors
  1. Ledder O1, 2
  2. Dolinger M3
  3. Dubinsky MC3
  4. Stein R4
  5. Vellanki S5
  6. Buckuk R1
  7. Fatima A6
  8. Suskind DL7
  9. Scarlett J7
  10. Roser D8
  11. Shouval DS9, 10
  12. Meyer G11
  13. Rios ZM11
  14. Pujolmuncunill G12
  15. Lozano A12
  16. Kolho KL13
  17. Rohani P14
  18. Hussey S15
  19. De Mejj T16
  20. Ayers T17
  21. Navaslopez VM18
  22. Turner D1, 2
  23. Tzivinikos C19
Show Affiliations
Authors Affiliations
  1. 1. Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel
  2. 2. Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel
  3. 3. Division of Pediatric Gastroenterology, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY, United States
  4. 4. Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, PA, United States
  5. 5. Division of Pediatric Gastroenterology, Hepatology and Nutrition, UT Southwestern Medical Center, Children's Medical Center, Dallas, TX, United States
  6. 6. Pediatric IBD Center, Beaumont Children's Hospital, Royal Oak, MI, United States
  7. 7. Division of Gastroenterology, Seattle Children's Hospital Inflammatory Bowel Disease Center, Seattle, WA, United States
  8. 8. Pediatric Department, Copenhagen University Hospital, Hvidovre, Denmark
  9. 9. Institute of Gastroenterology Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petach Tikva, Israel
  10. 10. Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
  11. 11. Division of Pediatric Gastroenterology, Nemours Children's Hospital, Wilmington, DE, United States
  12. 12. Pediatric Gastroenterology, Hepatology and Nutrition Department, Hospital Sant Joan de Deu, Barcelona, Spain
  13. 13. Pediatric Gastroenterology Department, Children's Hospital HUS, Helsinki University, Helsinki, Finland
  14. 14. Pediatric Gastroenterology and Hepatology Research Center, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran
  15. 15. Department of Paediatric Gastroenterology, Children's Health Ireland, Royal College of Surgeons of Ireland, University College Dublin, Dublin, Ireland
  16. 16. Department of Pediatric Gastroenterology, AG&M Research Institute, Amsterdam UMC, Amsterdam, Netherlands
  17. 17. Division of Pediatric Gastroenterology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, United States
  18. 18. Pediatric Gastroenterology and Nutrition Unit, Instituto de Investigacion Biomedica de Malaga, Hospital Regional Universitario de Malaga, Malaga, Spain
  19. 19. Paediatric Gastroenterology Department, Al Jalila Children's Specialty Hospital, Mohammed Bin Rashid University, Dubai, United Arab Emirates

Source: Inflammatory Bowel Diseases Published:2025


Abstract

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. © 2024 The Author(s). Published by Oxford University Press on behalf of Crohn's & Colitis Foundation. All rights reserved.