背景:类固醇难治性急性重度溃疡性结肠炎(ASUC)患者结肠切除术的风险最高。在可用的选项中,环孢素和英夫利昔单抗的疗效相似,但英夫利昔单抗是一种昂贵的药物,环孢素具有多种副作用,如肾损伤,神经毒性,和食欲不振。手术治疗通常与较高的发病率相关。较新的口服小分子如Janus激酶抑制剂是弥补这一差距的理想分子。托法替尼已经在中度至重度UC患者中进行了广泛的评估;然而,托法替尼治疗ASUC的数据有限.
方法:我们回顾性分析了2021年1月至2023年7月在我院腔胃肠病科住院的ASUC患者的资料。接受托法替尼治疗的ASUC患者被纳入研究。
结果:确定了8例ASUC患者对静脉内氢化可的松无反应,并接受了托法替尼治疗。平均年龄为39±15岁,女性占87.5%。中位病程为24个月(四分位距(IQR):12-120个月)。8名患者中有7名(87.5%)对口服托法替尼10mg,每天2次,治疗第5天。中位随访期为6个月(IQR:1-12个月)。一名患者需要结肠切除术,一名患者水痘带状疱疹重新激活,需要停止治疗。
结论:Tofacitinib是目前可用的类固醇难治性ASUC的挽救疗法的有吸引力的替代品;然而,长期疗效和风险仍有待探索。
BACKGROUND: Steroid-refractory acute severe ulcerative colitis (ASUC) patients are at the highest risk of colectomy. Among the available options, cyclosporine and infliximab have similar efficacy but infliximab is a costly drug and cyclosporine has multiple side effects like kidney injury, neurotoxicity, and dyselectrolytemia. Surgical management is often associated with higher morbidity. Newer oral small molecules like Janus kinase inhibitors are the ideal molecules to bridge the gap. Tofacitinib has already been extensively evaluated in patients with moderate to severe UC; however, data on ASUC treated by tofacitinib are limited.
METHODS: We retrospectively analyzed the data of patients with ASUC who were admitted to our hospital\'s luminal gastroenterology unit between January 2021 and July 2023. Patients with ASUC who were managed with tofacitinib were included in the study.
RESULTS: Eight patients with ASUC were identified who did not respond to intravenous hydrocortisone and were treated with tofacitinib. The mean age was 39 ± 15 years and 87.5% were female. The median duration of illness was 24 months (interquartile range (IQR): 12-120 months). Seven of eight patients (87.5%) responded to oral tofacitinib 10 mg twice a day by the fifth day of treatment. The median follow-up period was six months (IQR: 1-12 months). One patient required colectomy and one patient had varicella zoster reactivation requiring treatment discontinuation.
CONCLUSIONS: Tofacitinib is an attractive alternative to the currently available salvage therapy for steroid-refractory ASUC; however, long-term efficacy and risk remain to be explored.