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Real World Experience With Ustekinumab in Children and Young Adults at a Tertiary Care Pediatric Inflammatory Bowel Disease Center

Dayan, Judy R.; Dolinger, Michael; Benkov, Keith; Dunkin, David; Jossen, Jacqueline; Lai, Joanne; Phan, Becky L.; Pittman, Nanci; Dubinsky, Marla C.

Journal of Pediatric Gastroenterology and Nutrition: April 29, 2019 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPG.0000000000002362
Original Article: Gastroenterology: PDF Only
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Background: Ustekinumab is an effective therapy for Crohn disease currently approved for adults. Off-label use in the pediatric population is increasing, but its effectiveness in this age group has not been reported.

Aims: The aim of the study was to describe real-world experience with ustekinumab at a tertiary care pediatric inflammatory bowel disease (IBD) center.

Methods: As part of an ongoing observational cohort study of biologic-treated pediatric IBD patients initiated in October 2014, data on demographics, disease behavior, location and activity, treatment, and surgical history were collected for all patients receiving ustekinumab. Disease activity was assessed using the Harvey Bradshaw index or partial Mayo score. Primary outcome was steroid-free remission at 52 weeks. Descriptive statistics summarized the safety and efficacy outcomes, and univariate analyses were performed to examine associations of clinical characteristics with efficacy.

Results: Fifty-two children and young adults initiating ustekinumab were analyzed; 81% Crohn Disease, 8% ulcerative colitis, and 11% IBD-unspecified. Median [IQR] age at induction was 16.8 [14–18] years. Patients were followed for a minimum of 12 months. Most patients (81%) failed >1 anti-TNF, and 37% failed anti-TNF and vedolizumab; 10 patients were biologic-naïve. At week 52, 75% were still on ustekinumab, and 50% (bio-exposed) and 90% (bio-naïve) were in steroid-free remission. Two infusion reactions and neither serious adverse events nor serious infections were observed.

Conclusions: Our results suggest that ustekinumab is efficacious and safe in pediatric patients with IBD. Controlled clinical trial data are needed to confirm these observations.

Department of Pediatrics, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY.

Address correspondence and reprint requests to Marla C. Dubinsky, MD, 1 Gustave L Levy Place, Box 1134, New York, NY 10029 (e-mail: marla.dubinsky@mssm.edu).

Received 14 December, 2018

Accepted 16 March, 2019

No specific funding from any agency in the public, commercial, or not-for-profit sectors has been provided for the research.

M.C.D. has served as a consultant for Celgene, Boehringer Ingelheim, Genentech, Janssen, Pfizer, Takeda, and UCB, Abbvie, Protagonist, Valiant. The remaining authors have no conflict of interest.

© 2019 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,