Shimizu Hiromichi, Aonuma Yuko, Hibiya Shuji, Kawamoto Ami, Takenaka Kento, Fujii Toshimitsu, Saito Eiko, Nagahori Masakazu, Ohtsuka Kazuo, Okamoto Ryuichi
Department of Gastroenterology and Hepatology, Tokyo Medical and Dental University, Tokyo, Japan.
First Department of Internal Medicine, Faculty of Medicine, University of Yamanashi, Chuo, Japan.
Intest Res. 2024 Jul;22(3):369-377. doi: 10.5217/ir.2023.00194. Epub 2024 Jul 16.
BACKGROUND/AIMS: The efficacy and safety of tofacitinib for the treatment of refractory ulcerative colitis (UC) has been demonstrated in clinical trials. Although, a series of reports with real-world evidence of its short-term efficacy and safety profiles have already been published, reports of long-term real-world data have been limited. We aimed to show our 3-year evidence on the clinical use of tofacitinib for the treatment of UC, focusing on its efficacy and safety profiles.
A retrospective observational study was conducted on patients who started tofacitinib for active refractory UC at our hospital. The primary outcome was the retention rate until 156 weeks after initiating tofacitinib. The secondary outcomes were short-term efficacy at 4, 8, and 12 weeks; long-term efficacy at 52, 104, and 156 weeks; prognostic factors related to the cumulative retention rate; loss of response; and safety profile, including adverse events.
Forty-six patients who were able to be monitored for up to 156 weeks after tofacitinib initiation, were enrolled in this study. Continuation of tofacitinib was possible until 156 weeks in 54.3%, with > 50% response rates and > 40% remission rates. Among patients in whom response or remission was achieved and tofacitinib was deescalated after 8 weeks of induction treatment, 54.3% experienced relapse but were successfully rescued by and retained on reinduction treatment, except for 1 patient. No serious AEs were observed in the study.
Tofacitinib is effective and safe as long-term treatment in a refractory cohort of UC patients in real-world clinical practice.
背景/目的：托法替布治疗难治性溃疡性结肠炎（UC）的疗效和安全性已在临床试验中得到证实。尽管已经发表了一系列关于其短期疗效和安全性的真实世界证据报告，但长期真实世界数据的报告仍然有限。我们旨在展示我们关于托法替布治疗UC的3年临床使用证据，重点关注其疗效和安全性。
对我院开始使用托法替布治疗活动性难治性UC的患者进行回顾性观察研究。主要结局是开始使用托法替布后至156周的保留率。次要结局包括4、8和12周时的短期疗效；52、104和156周时的长期疗效；与累积保留率相关的预后因素；反应丧失；以及安全性，包括不良事件。
本研究纳入了46例在开始使用托法替布后能够被监测长达156周的患者。54.3%的患者能够持续使用托法替布至156周，缓解率>50%，应答率>40%。在诱导治疗8周后达到反应或缓解并降低托法替布剂量的患者中，54.3%经历了复发，但除1例患者外，其余患者通过再次诱导治疗成功挽救并继续使用该药物。研究中未观察到严重不良事件。
在真实世界的临床实践中，托法替布作为UC难治性患者的长期治疗有效且安全。
