Tofacitinib may be effective as a treatment of pediatric ulcerative colitis

by Simon Pan, Alex Chan

Originally published by 2 Minute Medicine® (view original article). Reused on AccessMedicine with permission.

1. Management of pediatric ulcerative colitis with tofacitinib successfully induced corticosteroid-free remission in 16% of patients at week 8 following treatment.

Evidence Rating Level: 2 (Good)

Tofacitinib, a non-selective inhibitor of Janus kinase (JAK) enzymes, was approved in 2018 for the treatment of moderate to severe ulcerative colitis in adults. However, it has not yet been approved for pediatric patients and there remains a lack of data regarding the efficacy and safety of tofacitinib in pediatric patients with ulcerative colitis. This multicentre, retrospective cohort study therefore sought to investigate the outcomes of the use of tofacitinib in pediatric ulcerative colitis. A total of 101 children were included in the analysis with a median follow-up of 24 weeks (IQR, 16-54 weeks) following initiation of tofacitinib, all of whom had received prior anti-tumour necrosis factor-α agents. The primary outcome was corticosteroid (CS)-free remission at week 8, while secondary outcomes included clinical response at week 8 and CS-free remission at all time points. 16% of patients (n = 16) achieved CS-free remission at week 8 following treatment, while 30% (n = 30) demonstrated clinical response but did not achieve CS-free remission. At week 24, 23% (n = 20) of 88 remaining patients achieved CS-free remission. No serious adverse events related to tofacitinib were reported in any patients. Overall, this study found that tofacitinib may be a safe and effective option in inducing CS-free remission in pediatric patients with ulcerative colitis previously refractory to other treatment modalities.

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