Long-term efficacy and safety of tofacitinib in patients with ulcerative colitis: 3-year results from a real-world study

Shimizu, Hiromichi; Aonuma, Yuko; Hibiya, Shuji; Kawamoto, Ami; Takenaka, Kento; Fujii, Toshimitsu; Saito, Eiko; Nagahori, Masakazu; Ohtsuka, Kazuo; Okamoto, Ryuichi

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Original Article

DOI: https://doi.org/10.5217/ir.2023.00194 [Epub ahead of print]
Published online July 16, 2024.

Long-term efficacy and safety of tofacitinib in patients with ulcerative colitis: 3-year results from a real-world study

Hiromichi Shimizu¹

¹Department of Gastroenterology and Hepatology, Tokyo Medical and Dental University, Tokyo, Japan
²First Department of Internal Medicine, Faculty of Medicine, University of Yamanashi, Chuo, Japan

Correspondence: Hiromichi Shimizu, Tel: +81-3-5803-5877, Fax: +81-3-5803-0263,
Email: hshigast@tmd.ac.jp

Received: 13 December 2023 • Revised: 12 March 2024 • Accepted: 22 March 2024

Abstract

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.
Methods
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.
Results
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.
Conclusions
Tofacitinib is effective and safe as long-term treatment in a refractory cohort of UC patients in real-world clinical practice.

Key Words: Ulcerative colitis, Efficacy, Real-world evidence, Safety, Tofacitinib