Immune checkpoint inhibitor (ICI) therapies have been established as the standard-of-care in various uro-oncological cancers. Immune-related adverse events (irAEs) are frequent, but their degree rarely leads to the discontinuation of immunotherapies. Unplanned permanent treatment discontinuation may negatively impact the outcomes of patients, but there are emerging data about a positive correlation between emergence of severe irAEs and therapeutic cancer responses. In this study, a retrospective analysis of patients treated for urothelial carcinoma (UC) with ICI-based immunotherapy was conducted. irAEs were classified according to the Common Terminology Criteria for Adverse Events (CTCAEs) and radiological responses according to the Response Evaluation Criteria In Solid Tumors (RECISTs). Out of 108 patients with metastatic urothelial cancer that underwent immunotherapy, 11 experienced a severe irAE that required permanent discontinuation of ICI therapy. The most frequent irAEs leading to discontinuation were hepatitis (n= 4), pneumonitis (n= 2), and gastritis or colitis (n= 2). Prior to discontinuation (R1), the radiological best response was complete remission (CR) in three patients, partial response (PR) in six, and stable disease (SD) in wo patients. After the discontinuation of ICI therapy (R2), the best responses were CR in six, PR in three, and SD in two patients. Following discontinuation, the majority of these patients showed a sustained treatment response, despite not receiving any cancer-specific treatment. The median time of response after discontinuation of ICI therapy was 26.0 (5.2–55.8) months. We propose accurate counseling and close follow-ups of patients following their discontinuation of ICI therapy due to irAEs, as responses can be durable and deep, and many patients do not require immediate subsequent therapies, even in urothelial cancer. More data are required to find predictors of the length of response to appropriately counsel patients.
免疫检查点抑制剂（ICI）疗法已成为多种泌尿系统肿瘤的标准治疗方案。免疫相关不良事件（irAEs）较为常见，但其严重程度很少导致免疫治疗中止。非计划性永久终止治疗可能对患者预后产生负面影响，但新近数据显示，严重irAEs的发生与抗肿瘤疗效之间存在正相关性。本研究对接受ICI为基础免疫治疗的尿路上皮癌（UC）患者进行回顾性分析。不良事件依据《不良事件通用术语标准》（CTCAEs）分级，影像学疗效依据《实体瘤疗效评价标准》（RECISTs）评估。在108例接受免疫治疗的转移性尿路上皮癌患者中，11例出现需永久停用ICI治疗的严重irAEs。导致治疗中止的最常见irAEs为肝炎（4例）、肺炎（2例）及胃炎或结肠炎（2例）。治疗中止前（R1），影像学最佳疗效为完全缓解（CR）3例、部分缓解（PR）6例、疾病稳定（SD）2例。ICI治疗中止后（R2），最佳疗效为CR 6例、PR 3例、SD 2例。尽管未接受任何肿瘤特异性治疗，多数患者在停药后仍维持治疗反应，其中位缓解持续时间为26.0（5.2–55.8）个月。我们建议对因irAEs停用ICI治疗的患者进行精准评估和密切随访，因其治疗反应可能具有持久性和深度，且许多患者（即使是尿路上皮癌患者）无需立即启动后续治疗。未来需要更多数据来寻找预测缓解持续时间的指标，以便为患者提供恰当的临床指导。
肿瘤(癌症)患者之家