Department of Pediatrics, Faculty of Medicine, Saga University, Saga, Japan.
Medicine (Baltimore). 2022 Nov 11;101(45):e31757. doi: 10.1097/MD.0000000000031757.
Tofacitinib is an orally administered selective Janus kinase inhibitor. Its efficacy and safety in adults with moderately to severely active ulcerative colitis (UC) have been evaluated in clinical trials; however, its efficacy in pediatric patients with UC is limited.
A 9-years-old boy was referred to our hospital with chief complaints of diarrhea and bloody stool for 2 months. Colonoscopy revealed total colitis-type UC. His pediatric UC activity index score was 40, indicating moderately active UC.
UC.
Vedolizumab, golimumab, and ustekinumab were introduced because of steroid-resistant refractory UC; however, none of these biologics were effective or the effect was short-lived. Therefore, tofacitinib was administered 5 mg twice a day.
The patient achieved UC remission after tofacitinib treatment, leading to maintained remission without adverse events.
To the best of our knowledge, this is the first pediatric case of moderately active UC that was successfully treated with tofacitinib in Japan. Tofacitinib is a safe drug for pediatric patients with moderately active UC. Even in steroid-dependent cases refractory to other biologics, tofacitinib can result in remission induction and maintenance effects. In children and adults, high-dose tofacitinib during induction therapy may be unnecessary to reduce adverse events.
托法替布是一种口服选择性 Janus 激酶抑制剂。其在中重度活动溃疡性结肠炎（UC）成年患者中的疗效和安全性已在临床试验中得到评估；然而，其在儿童 UC 患者中的疗效有限。
一名 9 岁男孩因腹泻和血性腹泻 2 个月就诊于我院。结肠镜检查显示全结肠炎型 UC。其儿童 UC 活动指数评分为 40，提示中重度 UC。
UC。
由于 UC 为激素抵抗性难治性，故引入了维多珠单抗、古塞奇尤单抗和乌司奴单抗；然而，这些生物制剂均无效或效果短暂。因此，给予托法替布 5mg，每日两次。
患者在接受托法替布治疗后 UC 缓解，且缓解持续，无不良反应。
据我们所知，这是日本首例成功用托法替布治疗中重度 UC 的儿科病例。托法替布是一种治疗中重度 UC 儿童患者的安全药物。即使在对其他生物制剂难治的依赖激素的病例中，托法替布也能诱导缓解并维持缓解。在儿童和成人中，诱导治疗时高剂量托法替布可能没有必要以减少不良反应。
