Division of Gastroenterology, Hepatology and Endoscopy, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Pfizer Inc, New York, NY, USA.
Inflamm Bowel Dis. 2024 Oct 3;30(10):1707-1713. doi: 10.1093/ibd/izad242.
Tofacitinib is an oral JAK inhibitor for the treatment of ulcerative colitis (UC). We assessed outcomes through 78 weeks of tofacitinib therapy for UC in a real-world setting.
This retrospective cohort study included adults initiating tofacitinib for UC from May 1, 2018, to April 1, 2021, at a large academic center in the United States. The primary outcome was steroid-free clinical remission at 78 (+/-4) weeks (SFCR 78; simple clinical colitis activity index ≤2 with no corticosteroid use within 30 days). The secondary outcome was tofacitinib discontinuation due to nonresponse (treatment persistence). Additional outcomes were endoscopic response/remission and adverse events (AEs).
Seventy-three patients initiated tofacitinib, with a median follow-up of 88 weeks. Among patients with available data, 31 of 60 (51.7%) achieved SFCR 78, 21 of 47 (44.7%) achieved endoscopic remission during follow-up, and 25 of 73 (34.2%) discontinued tofacitinib during follow-up due to nonresponse (including 11 patients who required colectomy). Nineteen AEs were reported among 15 patients during follow-up: shingles (n = 4, all without documented vaccinations), deep venous thrombosis (n = 2), elevated liver enzymes (n = 2), skin abscess (n = 2), pneumonia (n = 2), possible miscarriage (n = 2), norovirus (n = 1), COVID-19 (n = 1), lymphopenia (n = 1), Clostridioides difficile infection (n = 1), and heart block (n = 1). One patient discontinued therapy due to an AE (elevated liver enzymes), and no deaths occurred.
Tofacitinib treatment was effective in achieving SFCR for the majority of patients with UC through 78 weeks. Adverse events were consistent with the known safety profile of tofacitinib, and AEs requiring discontinuation were rare. Due to limitations regarding sample size, larger studies are needed to confirm these findings.
托法替尼是一种用于治疗溃疡性结肠炎（UC）的口服 JAK 抑制剂。我们在真实环境中评估了托法替尼治疗 UC 78 周的结果。
这是一项回顾性队列研究，纳入 2018 年 5 月 1 日至 2021 年 4 月 1 日期间在美国一家大型学术中心开始接受托法替尼治疗 UC 的成年人。主要结局是在 78（+/-4）周时达到无类固醇的临床缓解（SFCR78；简单临床结肠炎活动指数≤2，且在 30 天内未使用皮质类固醇）。次要结局是由于无应答（治疗持续时间）而停止使用托法替尼。其他结局是内镜缓解/缓解和不良事件（AE）。
73 例患者开始接受托法替尼治疗，中位随访时间为 88 周。在有可用数据的患者中，60 例中的 31 例（51.7%）达到 SFCR78，47 例中的 21 例（44.7%）在随访期间达到内镜缓解，73 例中的 25 例（34.2%）由于无应答而停止使用托法替尼（包括 11 例需要结肠切除术的患者）。15 例患者在随访期间报告了 19 例 AE：带状疱疹（n=4，均无记录疫苗接种）、深静脉血栓形成（n=2）、肝酶升高（n=2）、皮肤脓肿（n=2）、肺炎（n=2）、可能流产（n=2）、诺如病毒（n=1）、COVID-19（n=1）、淋巴细胞减少症（n=1）、艰难梭菌感染（n=1）和心脏阻滞（n=1）。1 例患者因 AE（肝酶升高）停止治疗，无死亡病例。
托法替尼治疗在 78 周内使大多数 UC 患者达到 SFCR，疗效显著。AE 与托法替尼已知的安全性特征一致，需要停药的 AE 罕见。由于样本量有限，需要更大的研究来证实这些发现。
