托法替布用于巴西溃疡性结肠炎的治疗：一项关于有效性和安全性的多中心观察性研究
Tofacitinib for ulcerative colitis in Brazil: a multicenter observational study on effectiveness and safety.
作者信息
Parra Rogério Serafim, de Sá Brito Fróes Renata, Magro Daniela Oliveira, da Costa Ferreira Sandro, de Mello Munique Kurtz, de Azevedo Matheus Freitas Cardoso, Damião Aderson Omar Mourão Cintra, de Sousa Carlos Alexandre, Barros Luísa Leite, de Miranda Maria Luiza Queiroz, Vieira Andrea, Sales Marcos Paulo Moraes, Zabot Gilmara Pandolfo, Cassol Ornella Sari, Tiburcio Alves Antonio José, Lubini Márcio, Machado Marta Brenner, Flores Cristina, Teixeira Fabio Vieira, Coy Claudio Saddy Rodrigues, Zaltman Cyrla, Chebli Liliana Andrade, Sassaki Ligia Yukie, Féres Omar, Chebli Júlio Maria Fonseca
机构信息
Department of Surgery and Anatomy, Ribeirão Preto Medical School, University of São Paulo. Ribeirão Preto, São Paulo, Brazil.
Gastromed, Department of Gastroenterology and Endoscopy, Rio de Janeiro, Brazil.
出版信息
BMC Gastroenterol. 2025 Mar 18;25(1):184. doi: 10.1186/s12876-025-03656-x.
AIM
To assess the real-life, long-term effectiveness and safety of tofacitinib in a large cohort of patients with refractory or difficult-to-treat ulcerative colitis (UC).
METHODS
This multicenter, retrospective, observational cohort study included patients with moderately to severely active UC who received tofacitinib for at least 8 weeks. Clinical remission and response, endoscopic response and remission, biochemical response and remission, steroid-free clinical remission, primary and secondary loss of response, drug discontinuation, the need for dose optimization, the need for colectomy, and adverse events were evaluated over up to 30 months.
RESULTS
We included 127 patients with UC, with a mean age of 40.3 ± 14.2 years; 58.2% were male, 75.6% had pancolitis, and 79.5% had previously failed at least one biological therapy, predominantly anti-TNF agents (70.1%). Clinical remission was observed in 31.5% of patients at weeks 12-16, 46.5% at 26 ± 4 weeks, and 37.0% at 1 year. Steroid-free clinical remission was achieved in 28.6%, 44.8%, and 37.1% of patients at the same time points, respectively. Biochemical remission was achieved in 33.6% of patients at 26 ± 4 weeks and 29.3% at 1 year. Endoscopic response and endoscopic remission within 1 year were observed in 46.0% and 15.3% of patients, respectively. Ten patients (7.9%) required colectomy, and 13 patients (10.2%) required hospitalization, all of whom had been previously exposed to biologics. The colectomy rate was significantly greater in patients with serum albumin levels ≤ 3.5 g/dL (21.4% vs. 4.1%, p = 0.013).
CONCLUSION
In this large, long-term real-world study involving patients with predominantly biologically refractory UC, tofacitinib effectively induced clinical remission and endoscopic improvement and prevented colectomy for more than 30 months, with a favorable safety profile. Notably, baseline hypoalbuminemia was associated with higher colectomy rates.
目的
评估托法替布在一大群难治性或难治疗的溃疡性结肠炎（UC）患者中的实际长期有效性和安全性。
方法
这项多中心、回顾性、观察性队列研究纳入了中度至重度活动性UC患者，这些患者接受托法替布治疗至少8周。在长达30个月的时间里评估临床缓解和反应、内镜反应和缓解、生化反应和缓解、无类固醇临床缓解、原发性和继发性反应丧失、药物停用、剂量优化需求、结肠切除术需求以及不良事件。
结果
我们纳入了127例UC患者，平均年龄为40.3±14.2岁；58.2%为男性，75.6%患有全结肠炎，79.5%此前至少一种生物治疗失败，主要是抗TNF药物（70.1%）。在第12 - 16周时，31.5%的患者实现临床缓解，在26±4周时为46.5%，在1年时为37.0%。在相同时间点，分别有28.6%、44.8%和37.1%的患者实现无类固醇临床缓解。在26±4周时，33.6%的患者实现生化缓解，在1年时为29.3%。1年内分别有46.0%和15.3%的患者观察到内镜反应和内镜缓解。10例患者（7.9%）需要进行结肠切除术，13例患者（10.2%）需要住院治疗，所有这些患者此前都曾接受过生物制剂治疗。血清白蛋白水平≤3.5g/dL的患者结肠切除率显著更高（21.4%对4.1%，p = 0.013）。
结论
在这项涉及主要为生物难治性UC患者的大型长期真实世界研究中，托法替布有效诱导临床缓解和内镜改善，并在超过30个月的时间里预防了结肠切除术，安全性良好。值得注意的是，基线低白蛋白血症与更高的结肠切除率相关。
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