Tofacitinib to Treat Severe Acute Refractory Colitis in a Teenager: Case Report and Review of the Literature

Abstract

Objectives: Acute severe colitis (ASC) may occur within 3 months of ulcerative colitis diagnosis in 9%–15% of children and the rate of colectomy is up to 40%–50% within 5 years after an ASC. The aim of this publication is to present recent and relevant data on the success of medical treatment with tofacitinib in ASC. Methods: We report a challenging case of a teenage boy with ASC at diagnosis and conduct a discussion after a review of the literature regarding the use of tofacitinib in inflammatory bowel disease, especially in pediatric patients and in ASC. Results: The patient was hospitalized for 10 weeks and was refractory to conventional therapies: intravenous corticosteroids, infliximab, methotrexate, and vedolizumab. He received 7 blood transfusions and also presented with a severe malnutrition requiring a total parenteral nutrition. Tofacitinib was considered as a medical last resort before colectomy and was started at week 8. Thirteen days after starting tofacitinib, he was asymptomatic and was discharged on tofacitinib as sole treatment. By week 9 of tofacitinib, a colonoscopy showed both endoscopic and histological remission. He has remained in clinical remission at 6-month follow-up. Conclusions: Tofacitinib may be an alternative medical treatment to avoid colectomy in ASC. It is a small molecule with a rapid onset and few severe adverse events. It has been used for ASC in adult patients, allowing to avoid colectomy in more than 60%. To our knowledge, this is one of the few pediatric patients with refractory ASC at initial diagnosis who responded to tofacitinib.

Recent developments in the assessment and management of inflammatory bowel disease in childhood: a narrative review

“…Currently there is no evidence to suggest that induction with alternative biological or small molecule therapies has a role in Crohn’s disease or ulcerative colitis in children ( 51 ). Despite this, data from adult studies would suggest that JAK-STAT inhibitors can be highly effective at induction in ulcerative colitis and some data from paediatric practice is observing highly effective rescue therapy in acute severe colitis ( 83 , 84 ).…”

“…There is a growing body of work, within adult practice, supporting the use of small molecule drugs (particularly tofacitinib) for acute colitis ( 4 , 83 , 84 , 95 ). Early reports in children mirror these findings, with Constant et al , reporting 8 of 11 children with acute severe colitis (ASC) being colectomy free at 90-day post tofacitinib initiation and 6 of 11 remaining so at a median 182-day follow-up ( 96 ).…”

The Role of Tofacitinib in the Treatment of Acute Severe Colitis in Children

“…To date, the data regarding the use in the pediatric setting is lacking, with our group reporting the first case. 23 Since then, we have treated thirteen patients with tofacitinib, six of whom received it for an ASC.…”