针对免疫检查点抑制剂（ICIs）引起的免疫相关不良事件（irAE）的治疗策略通常需要其他免疫抑制剂。托法替尼是一种快速起效的 JAK-STAT 抑制剂，已被证明对多种自身免疫性疾病有效。我们的目的是评估托法替布治疗癌症患者 irAE 的有效性和安全性。本研究回顾性纳入了在 6 家机构接受 ICI 并接受托法替布治疗 irAE 治疗的癌症患者。从电子病历中获得人口统计和临床特征。利用心肌肌钙蛋白 T (cTnT) 的纵向评估和临床评估来评估托法替布治疗 ICI 心肌炎患者的益处。还评估了总生存期 (OS)。本研究纳入了 53 名患者。从 irAE 发病到托法替布治疗的中位时间为 17（范围，2-186）天，托法替布治疗的中位持续时间为 52.5（范围，3-277）天。根据临床严重程度和类固醇反应性，纳入的患者被分为 3 组，包括 11 例危及生命的病例、30 例类固醇耐药病例和 12 例类固醇逐渐减量失败的病例。各组临床缓解率分别为54.5%、96.7%、100%（P<0.01）。托法替布耐受性良好，有 4 名患者 (7.5%) 发生感染事件。从 ICI 开始，总体中位 OS 为 16.1 (95% CI 7.8-26.9) 个月。托法替尼在经历 irAE 的患者中显示出有希望的临床疗效，特别是对类固醇治疗失败或在类固醇逐渐减少期间复发的患者。此外，最重要的是，托法替布在发生 irAE 的癌症患者中在毒性和抗肿瘤活性方面都表现出良好的安全性。未来的前瞻性研究是有必要的。© 2024。作者。

Treatment strategy against immune-related adverse events (irAEs) induced by immune checkpoint inhibitors (ICIs) frequently requires other immunosuppressive agents. Tofacitinib is a rapidly acting JAK-STAT inhibitor with proven efficacy in multiple autoimmune diseases. We aimed to evaluate the efficacy and safety of tofacitinib in the management of irAEs in cancer patients.Cancer patients who received ICIs and were treated with tofacitinib for the management of irAEs at 6 institutions were retrospectively included in this study. Demographic and clinical characteristics were obtained from electronic medical records. Longitudinal assessment of cardiac troponin T (cTnT) with clinical assessment was utilized to evaluate the benefit of tofacitinib treatment in patients with ICI myocarditis. Overall survival (OS) was also assessed.Fifty-three patients were included in this study. The median time from irAE onset to tofacitinib therapy was 17 (range, 2-186) days and the median duration of tofacitinib treatment was 52.5 (range, 3-277) days. Enrolled patients were subdivided into 3 groups based on clinical severity and steroid responsiveness including 11 life-threatening cases, 30 steroid-resistant cases, and 12 cases with steroid taper failure. Clinical remission rate in each group was 54.5%, 96.7%, and 100%, respectively (P < 0.01). Tofacitinib was well-tolerated with 4 patients (7.5%) developing infectious events. From the ICI initiation, the overall median OS was 16.1 (95% CI 7.8-26.9) months.Tofacitinib showed promising clinical efficacy in patients experiencing irAEs, particularly in patients who failed to respond to steroids or experienced relapse during steroid tapering. Moreover, and most importantly, tofacitinib exhibited a favorable safety profile in cancer patients developing irAEs in terms of both toxicity and anti-tumor activity. Future prospective studies are warranted.© 2024. The Author(s).