Intest Res.  2024 Jul;22(3):369-377. 10.5217/ir.2023.00194.

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

• Shimizu H
• Aonuma Y
• Hibiya S
• Kawamoto A
• Takenaka K
• Fujii T
• Saito E
• Nagahori M
• Ohtsuka K
• Okamoto R

• KMID: 2558196
• DOI: http://doi.org/10.5217/ir.2023.00194

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.

Keyword

Ulcerative colitis; Efficacy; Real-world evidence; Safety; Tofacitinib

Figure

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  Fig. 1. Survival curve of patients who maintained tofacitinib treatment over time up to 156 weeks after initiation of tofacitinib treatment (n=46).

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  Fig. 2. Efficacy of tofacitinib (n=25). Tofacitinib treatment non-failure patients showed significantly improved partial Mayo score from as early as 4 weeks after tofacitinib initiation, which continued until 156 weeks. aWilcoxon test: P<0.001 compared to week 0.

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  Fig. 3. Time-series data of short-term and long-term efficacy of tofacitinib (n=46). Remission rate was 31.1% at 4 weeks, 37.0% at 8 weeks, and 43.5% at 12 weeks. Remission rate continued to exceed 40% from 12 through 156 weeks. Response rate was 44.4% at 4 weeks and the rate continued to exceed over 50% from 8 weeks through 156 weeks (n=46).

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