Tofacitinib Therapy in Children With Pediatric-onset Medical
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.

This study included subjects ages 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.

--21 subjects, 18 with ulcerative colitis or indeterminate IBD, received tofacitinib.

--At the end of the 12-week induction period, 9 out of 21 subjects showed clinical response and 7 out of 21 were in steroid-free remission.

--Of evaluable subjects at 52 weeks, 7 out of 17 showed clinical response and were in steroid-free remission.

--Of those remaining on tofacitinib at 1 year, none required concomitant systemic corticosteroids.

--Tofacitinib was discontinued in 8 subjects because of refractory disease, including 8 who ultimately underwent colectomy, and in 1 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.

Conclusively, there is minimal experience with tofacitinib in the treatment of pediatric IBD. Tofacitinib induced a quick clinical response with long-term efficacy in approximately half of the individuals in this cohort. This study adds to the growing body of evidence supporting tofacitinib's efficacy and safety as part of the therapy paradigm for young people with moderate-to-severe IBD.