Immunotherapy has been used in localized urothelial carcinoma for decades, especially in the treatment of superficial disease, in which instillation of BCG is a commonly used treatment option. Clinical investigations based on new insights into the immunogenic potential of metastatic urothelial carcinoma have led to the accelerated FDA approval of the immune checkpoint inhibitors atezolizumab, nivolumab, durvalumab, avelumab, and pembrolizumab. Preliminary findings suggest additional benefits of combinations of immunotherapeutic agents as a future treatment approach in metastatic urothelial carcinoma. Treatment experience with immunotherapy suggests that these drugs are associated with a unique spectrum of immune-related adverse events and specific immune-related patterns of response, including cases of pseudoprogression, which could impede the optimal use of immune checkpoint inhibitors in the clinic. Appropriate management of immune-related adverse events and a greater awareness of immune-mediated response patterns will help to inform treatment decisions and improve patient outcomes; predictive biomarkers of response might facilitate selection of patients who are most likely to respond to and benefit from these exciting new treatments.

免疫疗法已在局部尿路上皮癌中使用了数十年，特别是在浅表疾病的治疗中，其中滴注BCG是一种常用的治疗选择。基于对转移性尿路上皮癌的免疫原性潜力的新见识的临床研究已导致FDA加快了对免疫检查点抑制剂atezolizumab，nivolumab，durvalumab，avelumab和pembrolizumab的FDA批准。初步发现表明，将免疫治疗剂组合作为转移性尿路上皮癌的未来治疗方法还有其他好处。免疫疗法的治疗经验表明，这些药物与一系列独特的免疫相关不良事件和特定的免疫相关反应模式有关，包括伪进展，这可能会阻碍临床中免疫检查点抑制剂的最佳使用。对免疫相关不良事件的适当管理以及对免疫介导反应模式的更深入了解将有助于为治疗决策提供依据并改善患者预后；预测性反应生物标志物可能有助于选择最有可能对这些令人振奋的新疗法产生反应并从中受益的患者。