Inflamm Bowel Dis. 2025 Feb 6;31(2):425-431. doi: 10.1093/ibd/izae112.

Oren Ledder ^1 2, Michael Dolinger ³, Marla C Dubinsky ³, Ronen Stein ⁴, Srisindu Vellanki ⁵, Rachel Buckuk ¹, Ayesha Fatima ⁶, David L Suskind ⁷, Jarrad Scarlett ⁷, Dennis Röser ⁸, Dror S Shouval ^9 10, Gabriele Meyer ¹¹, Zarela Molle Rios ¹¹, Gemma Pujol-Muncunill ¹², Anna Lozano ¹², Kaija-Leena Kolho ¹³, Pejman Rohani ¹⁴, Seamus Hussey ¹⁵, Tim de Mejj ¹⁶, Travis Ayers ¹⁷, Victor Manuel Navas-López ¹⁸, Dan Turner ^1 2, Christos Tzivinikos ¹⁹

Author information

¹Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel.

²Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.

³Division of Pediatric Gastroenterology, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

⁴Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, PA, USA.

⁵Division of Pediatric Gastroenterology, Hepatology and Nutrition, UT Southwestern Medical Center and Children's Medical Center, Dallas, TX, USA.

⁶Pediatric IBD Center, Beaumont Children's Hospital, Royal Oak, MI, USA.

⁷Division of Gastroenterology, Seattle Children's Hospital Inflammatory Bowel Disease Center, Seattle, WA, USA.

⁸Pediatric Department, Copenhagen University Hospital, Hvidovre, Denmark.

⁹Institute of Gastroenterology Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petach Tikva.

¹⁰Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

¹¹Division of Pediatric Gastroenterology, Nemours Children's Hospital, Wilmington, DE, USA.

¹²Pediatric Gastroenterology, Hepatology and Nutrition Department, Hospital Sant Joan de Deu, Barcelona, Spain.

¹³Pediatric Gastroenterology Department, Children's Hospital HUS, Helsinki University, Helsinki, Finland.

¹⁴Pediatric Gastroenterology and Hepatology Research Center, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.

¹⁵Department of Paediatric Gastroenterology, Children's Health Ireland, Royal College of Surgeons of Ireland and University College Dublin, Dublin, Ireland.

¹⁶Department of Pediatric Gastroenterology, AG&M Research Institute, Amsterdam UMC, Amsterdam, the Netherlands.

¹⁷Division of Pediatric Gastroenterology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.

¹⁸Pediatric Gastroenterology and Nutrition Unit, Instituto de Investigación Biomédica de Málaga, Hospital Regional Universitario de Málaga, Málaga, Spain.

¹⁹Paediatric Gastroenterology Department, Al Jalila Children's Specialty Hospital, Mohammed Bin Rashid University, Dubai, United Arab Emirates.

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.