BACKGROUND
Ulcerative colitis (UC) is a chronic autoimmune inflammatory disease of the colon that infrequently affects children. The disease requires immunosuppressive therapy to achieve remission and keep the disease in remission. Currently, many
therapies are approved for use in pediatric patients with UC, including
steroid,
5-aminosalicylic acid (5-ASA),
azathioprine, and
biologic therapy with anti-
tumor necrosis factor (
TNF) inhibitors. Despite their efficacy, many patients have refractory severe disease that fails
therapy and may require surgical interventions. Recently, the small molecule
Janus Kinase (
JAK) inhibitor tofacitinib has been approved for moderate to severe UC that fails
biologic therapy in adults. However, the safety and efficacy of this
drug has not been tested in pediatric UC patients. CASE REPORT We describe a case of a 13-year-old girl with 2-year history of severe UC who had secondary loss response to both
infliximab and
adalimumab over 2 years, despite adequate trough serum
drug levels and the concomitant use of
azathioprine. She was also dependent on
steroid to control
her disease. Infectious work-ups were always negative for infectious organisms. She was then successfully treated with
tofacitinib 5 mg orally twice daily. She went into complete clinical, endoscopic, and
steroid-free remission. CONCLUSIONS This case report highlights the safety and efficacy of
tofacitinib in pediatric patients with severe refractory UC, potentially avoiding
proctocolectomy in this young patient population. Future research should study the role of
tofacitinib in patients with moderate to severe UC in children.