日本溃疡性结肠炎患者中托法替布的上市后监测：安全性和有效性数据的最终报告
Post-marketing surveillance of tofacitinib in patients with ulcerative colitis in Japan: a final report of safety and effectiveness data.
作者信息
Matsuoka Katsuyoshi, Motoya Satoshi, Yamamoto Takayuki, Matsuura Minoru, Fujii Toshimitsu, Shinzaki Shinichiro, Mikami Yohei, Arai Shoko, Oshima Junichi, Endo Yutaka, Yuasa Hirotoshi, Hoshi Masato, Sato Keiko, Hisamatsu Tadakazu
机构信息
Division of Gastroenterology and Hepatology, Department of Internal Medicine, Toho University Sakura Medical Center, Chiba, Japan.
Clinical Epidemiology Committee of the Japanese Society for Inflammatory Bowel Disease, Shinjuku-ku, Tokyo, Japan.
出版信息
J Gastroenterol. 2025 Apr 21. doi: 10.1007/s00535-025-02249-5.
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.
背景
我们展示了一项关于托法替布在日本溃疡性结肠炎（UC）患者中的上市后监测（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）。登记了日本所有接受托法替布治疗的UC患者（观察期60周）。记录不良事件（AE）。根据方案，确定了几种临床上重要/潜在风险的AE；所有治疗期数据用于计算发病率（IR；发生事件的独特患者数/100患者年[PY]暴露）。评估有效性（部分/总梅奥评分），采用末次观察值结转法对缺失数据进行插补。
结果
总体而言，共纳入2043例患者（安全性分析集：n = 1982/有效性分析集：n = 1969）。排除了来自两家医院的13例患者的数据，这两家医院未获得发表同意，因此不允许发表。分别有33.4%和5.2%的患者观察到AE和严重AE；发生1例死亡（肠脓肿）。带状疱疹（HZ；非严重和严重）是报告最多的感染（n = 92 [IR 5.93/100 PY，95%置信区间4.78，7.27]）。严重感染、恶性肿瘤、心血管和静脉血栓栓塞事件的IR分别为1.51/100 PY、0.62/100 PY、0.13/100 PY和0.31/100 PY。总体而言，52.4%的患者停止治疗，主要原因是临床反应不佳（48.9%）。在第60周时，1151/1969例患者（58.5%）实现了部分梅奥评分缓解。
结论
总体安全性概况与来自日本、全球的PMS报告以及托法替布UC临床项目中的托法替布数据总体相当。然而，HZ的IR高于托法替布UC临床项目。托法替布的有效性与托法替布UC临床项目的数据一致。
临床试验
政府编号：NCT03643211。
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