Retrospective Study
Copyright ©The Author(s) 2024. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Apr 7, 2024; 30(13): 1871-1886
Published online Apr 7, 2024. doi: 10.3748/wjg.v30.i13.1871
Real-world efficacy and safety of tofacitinib treatment in Asian patients with ulcerative colitis
Kentaro Kojima, Kenji Watanabe, Mikio Kawai, Soichi Yagi, Koji Kaku, Maiko Ikenouchi, Toshiyuki Sato, Koji Kamikozuru, Yoko Yokoyama, Tetsuya Takagawa, Masahito Shimizu, Shinichiro Shinzaki
Kentaro Kojima, Kenji Watanabe, Mikio Kawai, Soichi Yagi, Koji Kaku, Maiko Ikenouchi, Toshiyuki Sato, Koji Kamikozuru, Yoko Yokoyama, Shinichiro Shinzaki, Department of Gastroenterology, Hyogo Medical University, Nishinomiya 663-8501, Japan
Kentaro Kojima, Masahito Shimizu, Department of Gastroenterology, Gifu University Graduate School of Medicine, Gifu 501-1194, Japan
Kenji Watanabe, Department of Internal Medicine for Inflammatory Bowel Disease, The University of Toyama, Toyama 930-0194, Japan
Tetsuya Takagawa, Center for Clinical Research and Education, Hyogo Medical University, Nishinomiya 663-8501, Japan
Author contributions: Kojima K and Watanabe K participated in the conception and design of the study and were involved in the acquisition, analysis, or interpretation of data; Kojima K wrote the manuscript; Watanabe K and Shinzaki S accessed and verified the study data. All authors critically reviewed and provided final approval of the manuscript; all authors were responsible for the decision to submit the manuscript for publication.
Institutional review board statement: This investigation was approved by the Institutional Ethics Committee of Hyogo Medical University Hospital, No. 3030.
Informed consent statement: The need for patient consent was waived due to the retrospective nature of the study.
Conflict-of-interest statement: Watanabe K received honoraria and had expenses paid to attend or give a presentation or advice at a meeting for the following companies: AbbVie GK, EA Pharma Co., Ltd., Pfizer Japan Inc., Takeda Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Kyorin Pharmaceutical Co., Ltd., Mochida Pharmaceutical Co., Ltd., Kissei Pharmaceutical Co., Ltd.; received research grants from EA Pharma Co., Ltd., Takeda Pharmaceutical Co., Ltd., EP-CRSU Co., Ltd., received scholarship grants from AbbVie GK, EA Pharma Co., Ltd., Mitsubishi Tanabe Pharma Corporation, JIMRO Co., Ltd., Nippon Kayaku Co., Ltd.; and has been an endowed chair for AbbVie GK, EA Pharma Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Zeria Pharmaceutical Co., Ltd., JIMRO Co., Ltd., Otsuka Pharmaceutical Factory, Inc., Asahi Kasei Medical Co., Ltd., Mochida Pharmaceutical Co., Ltd. SS received honoraria and had expenses paid to attend or give a presentation or advice at meetings for AbbVie GK, EA Pharma Co., Ltd., Pfizer Japan Inc., Takeda Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Kyorin Pharmaceutical Co., Ltd., Mochida Pharmaceutical Co., Ltd., Kissei Pharmaceutical Co., Ltd., Janssen Pharmaceutical K.K., Nippon Kayaku Co., Ltd., Gilead Sciences, JIMRO Co., Ltd., Nippon Kayaku Co., Zeria Pharmaceutical Co., Ltd., Alfresa Pharma Corporation, Astra Zeneka K.K., Eisai Co., Ltd., Sekisui Medical Co., Ltd. And received research grants from Sekisui Medical Co., Ltd.
Data sharing statement: No additional data are available.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Kenji Watanabe, FACG, MD, PhD, Professor, Department of Gastroenterology, Hyogo Medical University, 1-1, Mukogawa-cho, Nishinomiya City, Hyogo Prefecture, Nishinomiya 663-8501, Japan. kenjiw@med.u-toyama.ac.jp
Received: January 9, 2024
Peer-review started: January 9, 2024
First decision: January 17, 2024
Revised: January 30, 2024
Accepted: March 13, 2024
Article in press: March 13, 2024
Published online: April 7, 2024
Processing time: 84 Days and 16.6 Hours
Abstract
BACKGROUND

Real-world data on tofacitinib (TOF) covering a period of more than 1 year for a sufficient number of Asian patients with ulcerative colitis (UC) are scarce.

AIM

To investigate the long-term efficacy and safety of TOF treatment for UC, including clinical issues.

METHODS

We performed a retrospective single-center observational analysis of 111 UC patients administered TOF at Hyogo Medical University as a tertiary inflammatory bowel disease center. All consecutive UC patients who received TOF between May 2018 and February 2020 were enrolled. Patients were followed up until August 2020. The primary outcome was the clinical response rate at week 8. Secondary outcomes included clinical remission at week 8, cumulative persistence rate of TOF administration, colectomy-free survival, relapse after tapering of TOF and predictors of clinical response at week 8 and week 48.

RESULTS

The clinical response and remission rates were 66.3% and 50.5% at week 8, and 47.1% and 43.5% at week 48, respectively. The overall cumulative clinical remission rate was 61.7% at week 48 and history of anti-tumor necrosis factor-alpha (TNF-α) agents use had no influence (P = 0.25). The cumulative TOF persistence rate at week 48 was significantly lower in patients without clinical remission than in those with remission at week 8 (30.9% vs 88.1%; P < 0.001). Baseline partial Mayo Score was significantly lower in responders vs non-responders at week 8 (odds ratio: 0.61, 95% confidence interval: 0.45-0.82, P = 0.001). Relapse occurred in 45.7% of patients after TOF tapering, and 85.7% of patients responded within 4 wk after re-increase. All 6 patients with herpes zoster (HZ) developed the infection after achieving remission by TOF.

CONCLUSION

TOF was more effective in UC patients with mild activity at baseline and its efficacy was not affected by previous treatment with anti-TNF-α agents. Most relapsed patients responded again after re-increase of TOF and nearly half relapsed after tapering off TOF. Special attention is needed for tapering and HZ.

Keywords: Ulcerative colitis; Tofacitinib; Janus kinase inhibitor; Real-world; Biologics

Core Tip: This is a retrospective single-center observational study to investigate the long-term efficacy and safety of tofacitinib (TOF) treatment for ulcerative colitis (UC), including clinical issues. TOF is more effective in low-activity UC patients and its efficacy is not affected by previous treatment with anti-tumor necrosis factor-alpha agents. Most patients in the clinical remission group at week 8 could continue TOF over a long follow-up period. Most relapsed patients responded again after re-increase of TOF and nearly half relapsed after tapering off TOF. Although most patients continue TOF without severe adverse events, careful monitoring for herpes zoster is necessary.