Post-marketing surveillance of tofacitinib in patients with ulcerative colitis in Japan: a final report of safety and effectiveness data.

IF 6.9 2区医学 Q1 GASTROENTEROLOGY & HEPATOLOGY

Journal of Gastroenterology Pub Date : 2025-04-21 DOI:10.1007/s00535-025-02249-5

Katsuyoshi Matsuoka, Satoshi Motoya, Takayuki Yamamoto, Minoru Matsuura, Toshimitsu Fujii, Shinichiro Shinzaki, Yohei Mikami, Shoko Arai, Junichi Oshima, Yutaka Endo, Hirotoshi Yuasa, Masato Hoshi, Keiko Sato, Tadakazu Hisamatsu

{"title":"Post-marketing surveillance of tofacitinib in patients with ulcerative colitis in Japan: a final report of safety and effectiveness data.","authors":"Katsuyoshi Matsuoka, Satoshi Motoya, Takayuki Yamamoto, Minoru Matsuura, Toshimitsu Fujii, Shinichiro Shinzaki, Yohei Mikami, Shoko Arai, Junichi Oshima, Yutaka Endo, Hirotoshi Yuasa, Masato Hoshi, Keiko Sato, Tadakazu Hisamatsu","doi":"10.1007/s00535-025-02249-5","DOIUrl":null,"url":null,"abstract":"Background: We present the final analysis of a tofacitinib post-marketing surveillance (PMS) study in Japanese patients with ulcerative colitis (UC).Methods: Safety/effectiveness data were evaluated (through Sept/30/2022). All patients with UC in Japan receiving tofacitinib were registered (60-week observation period). Adverse events (AEs) were recorded. Per protocol, several AEs were identified as clinically important/potential risks; all treatment-period data were used to calculate incidence rates (IRs; unique patients with events/100 patient-years [PY] of exposure). Effectiveness was assessed (partial/total Mayo score), with last observation carried forward for imputation of missing data.Results: Overall, 2043 patients were enrolled (safety analysis set: n = 1982/effectiveness analysis set: n = 1969). Data were excluded for 13 patients from two hospitals from which consent was not obtained for publication and which, therefore, were not permitted for publication. AEs and serious AEs were observed in 33.4% and 5.2% of patients, respectively; one death occurred (intestinal abscess). Herpes zoster (HZ; non-serious and serious) was the most reported infection (n = 92 [IR 5.93/100 PY, 95% confidence interval 4.78, 7.27]). Serious infection, malignancy, cardiovascular and venous thromboembolic events IRs were 1.51/100 PY, 0.62/100 PY, 0.13/100 PY, and 0.31/100 PY, respectively. Overall, 52.4% of patients discontinued treatment, mostly due to inadequate clinical response (48.9%). At Week 60, 1151/1969 patients (58.5%) achieved partial Mayo score remission.Conclusion: The overall safety profile was generally comparable with tofacitinib data from PMS reports from Japan, worldwide and the tofacitinib UC clinical program. However, HZ IR was higher than in the tofacitinib UC clinical program. Tofacitinib effectiveness was consistent with data from the tofacitinib UC clinical program.Clinicaltrials: GOV: NCT03643211.","PeriodicalId":16059,"journal":{"name":"Journal of Gastroenterology","volume":" ","pages":""},"PeriodicalIF":6.9000,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Gastroenterology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s00535-025-02249-5","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"GASTROENTEROLOGY & HEPATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Background: We present the final analysis of a tofacitinib post-marketing surveillance (PMS) study in Japanese patients with ulcerative colitis (UC).

Methods: Safety/effectiveness data were evaluated (through Sept/30/2022). All patients with UC in Japan receiving tofacitinib were registered (60-week observation period). Adverse events (AEs) were recorded. Per protocol, several AEs were identified as clinically important/potential risks; all treatment-period data were used to calculate incidence rates (IRs; unique patients with events/100 patient-years [PY] of exposure). Effectiveness was assessed (partial/total Mayo score), with last observation carried forward for imputation of missing data.

Results: Overall, 2043 patients were enrolled (safety analysis set: n = 1982/effectiveness analysis set: n = 1969). Data were excluded for 13 patients from two hospitals from which consent was not obtained for publication and which, therefore, were not permitted for publication. AEs and serious AEs were observed in 33.4% and 5.2% of patients, respectively; one death occurred (intestinal abscess). Herpes zoster (HZ; non-serious and serious) was the most reported infection (n = 92 [IR 5.93/100 PY, 95% confidence interval 4.78, 7.27]). Serious infection, malignancy, cardiovascular and venous thromboembolic events IRs were 1.51/100 PY, 0.62/100 PY, 0.13/100 PY, and 0.31/100 PY, respectively. Overall, 52.4% of patients discontinued treatment, mostly due to inadequate clinical response (48.9%). At Week 60, 1151/1969 patients (58.5%) achieved partial Mayo score remission.

Conclusion: The overall safety profile was generally comparable with tofacitinib data from PMS reports from Japan, worldwide and the tofacitinib UC clinical program. However, HZ IR was higher than in the tofacitinib UC clinical program. Tofacitinib effectiveness was consistent with data from the tofacitinib UC clinical program.

Clinicaltrials: GOV: NCT03643211.

查看原文本刊更多论文

托法替尼在日本溃疡性结肠炎患者的上市后监测：安全性和有效性数据的最终报告

背景：我们提出了一项针对日本溃疡性结肠炎（UC）患者的托法替尼上市后监测（PMS）研究的最终分析。方法：评估安全性/有效性数据（截至2022年9月30日）。所有接受托法替尼治疗的日本UC患者均被登记（60周观察期）。记录不良事件（ae）。根据每个方案，几个ae被确定为临床重要/潜在风险；所有治疗期数据用于计算发病率(IRs；事件的独特患者/100患者年[PY]的暴露)。评估有效性（部分/总Mayo评分），并将最后一次观察结转，以插入缺失数据。结果：共纳入2043例患者（安全性分析组：n = 1982/有效性分析组：n = 1969）。排除了来自两家医院的13名患者的数据，这两家医院未获得发表的同意，因此不允许发表。不良反应发生率为33.4%，严重不良反应发生率为5.2%；1例死亡（肠脓肿）。带状疱疹；非严重和严重)感染最多（n = 92 [IR 5.93/100 PY， 95%可信区间4.78,7.27]）。严重感染、恶性、心血管和静脉血栓栓塞事件发生率分别为1.51/100 PY、0.62/100 PY、0.13/100 PY、0.31/100 PY。总体而言，52.4%的患者停止治疗，主要是由于临床反应不足（48.9%）。在第60周，1151/1969例患者（58.5%）获得部分梅奥评分缓解。结论：tofacitinib的总体安全性与来自日本、全球和tofacitinib UC临床项目的PMS报告的tofacitinib数据大致相当。然而，赫兹IR高于托法替尼UC临床项目。托法替尼的有效性与托法替尼UC临床项目的数据一致。临床试验：GOV: NCT03643211。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Journal of Gastroenterology 医学-胃肠肝病学

CiteScore

12.20

自引率

1.60%

发文量

审稿时长

4-8 weeks

期刊介绍： The Journal of Gastroenterology, which is the official publication of the Japanese Society of Gastroenterology, publishes Original Articles (Alimentary Tract/Liver, Pancreas, and Biliary Tract), Review Articles, Letters to the Editors and other articles on all aspects of the field of gastroenterology. Significant contributions relating to basic research, theory, and practice are welcomed. These publications are designed to disseminate knowledge in this field to a worldwide audience, and accordingly, its editorial board has an international membership.