Tofacitinib for a Child with Refractory Steroid-Dependent Ulcerative Colitis: A Case Report and Review of the Literature

Refaa Alajmi 1, Munirah Alabdulhadi 2, Ali A Alali 2, Mohammad Shehab 2

Affiliations

12 November 2021

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doi: 10.12659/AJCR.934460

Abstract

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.

Conflict of interest statement

Conflict of interest: None declared

Figures

Figure 1. This graph represents the body mass index (BMI-for-age) based on the World Health Organization method. (A) At the onset of the disease with BMI of 17.3 kg/m2; (B) When she was on infliximab 10 mg/kg every 8 weeks and azathioprine 2.5 mg/kg), her BMI was 19.3 kg/m2. (C) When she relapsed after failing infliximab with BMI of 17.3 kg/m2. (D) The current body mass index BMI is 21.2 kg/m2.

Figure 1. This graph represents the body mass index (BMI-for-age) based on the World Health Organization method. (A) At the onset of the disease with BMI of 17.3 kg/m2; (B) When she was on infliximab 10 mg/kg every 8 weeks and azathioprine 2.5 mg/kg), her BMI was 19.3 kg/m2. (C) When she relapsed after failing infliximab with BMI of 17.3 kg/m2. (D) The current body mass index BMI is 21.2 kg/m2.

Figure 2. PicturesA and B showing the rectum and sigmoid colon, respectively, before starting tofacitinib, with severe ulcerations, inflammation, loss of vascularity, pseudopolyps, and severely deformed lumen. Pictures C and D showing the rectum and sigmoid, respectively, 9 months after starting tofacitinib, with no ulcerations or erosions except for mild patchy erythema.

Figure 2. PicturesA and B showing the rectum and sigmoid colon, respectively, before starting tofacitinib, with severe ulcerations, inflammation, loss of vascularity, pseudopolyps, and severely deformed lumen. Pictures C and D showing the rectum and sigmoid, respectively, 9 months after starting tofacitinib, with no ulcerations or erosions except for mild patchy erythema.

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