Tofacitinib Therapy in Children and Young Adults with Pediatric-Onset Medically-Refractory Inflammatory Bowel Disease

Objectives: Tofacitinib, a selective Janus kinase inhibitor, effectively induces and maintains remission in adults with inflammatory bowel disease (IBD), but data are limited in children. This study aimed to evaluate the efficacy and safety of tofacitinib for medically-refractory pediatric-onset IBD.

Methods: This single-center retrospective study included subjects aged 21 years and younger who started tofacitinib for medically-refractory IBD. Clinical activity indices, clinical response, steroid-free remission, biochemical response, and adverse events (AEs) were evaluated over 52 weeks.

Results: Twenty-one subjects, 18 with ulcerative colitis or indeterminate IBD, received tofacitinib. At the end of the 12 week induction period, 9/21 (42.9%) subjects showed clinical response and 7/21 (33.3%) were in steroid-free remission. Of evaluable subjects at 52 weeks, 7/17 (41.2%) showed clinical response and were in steroid-free remission. Of those remaining on tofacitinib at one year, none required concomitant systemic corticosteroids. Tofacitinib was discontinued in eight subjects due to refractory disease, including six who ultimately underwent colectomy, and in one subject who developed a sterile intra-abdominal abscess. There were no instances of thrombi, zoster reactivation, or clinically-significant hyperlipidemia, all of which were AEs of interest.

Conclusions: There is limited experience with tofacitinib in pediatric IBD. In this cohort, tofacitinib induced rapid clinical response with sustained efficacy in nearly half of subjects. This study provides encouraging evidence for the efficacy and safety of tofacitinib as part of the treatment paradigm for young individuals with moderate-to-severe IBD. Larger, well-powered, prospective studies are warranted.

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