Background/Objectives: Urothelial carcinoma (UC) treatment has been transformed by immunotherapy and antibody-drug conjugates (ADCs). This review evaluates the current evidence for these approaches and identifies future directions. Methods: We conducted a structured review of clinical trials, meta-analyses, and guidelines published until early 2025. Results: Immune checkpoint inhibitors have established benefits across multiple settings: post-platinum therapy (pembrolizumab, nivolumab), maintenance therapy (avelumab), adjuvant settings for high-risk muscle-invasive disease (nivolumab), and BCG-unresponsive non-muscle-invasive disease (pembrolizumab). Enfortumab vedotin (targeting Nectin-4) has proven effective in post-platinum/post-immunotherapy. Most significantly, enfortumab vedotin plus pembrolizumab has redefined first-line treatment with unprecedented survival benefits (median OS 31.5 months vs. 16.1 months with chemotherapy; HR 0.47), and nivolumab plus gemcitabine-cisplatin improved outcomes in cisplatin-eligible patients. Key challenges include managing unique toxicity profiles, optimizing treatment sequencing, and developing reliable biomarkers. Conclusions: Combination approaches offer the most promising path forward, with future research needed on resistance mechanisms, biomarker development, and expanding these therapies to earlier disease stages.
**背景/目的：** 尿路上皮癌的治疗因免疫疗法和抗体药物偶联物的应用而发生变革。本综述评估了这些方法的现有证据并指出了未来方向。 **方法：** 我们对截至2025年初发表的临床试验、荟萃分析和指南进行了系统性回顾。 **结果：** 免疫检查点抑制剂在多种治疗场景中已确立其获益：铂类药物治疗后（帕博利珠单抗、纳武利尤单抗）、维持治疗（阿维鲁单抗）、高危肌层浸润性疾病的辅助治疗（纳武利尤单抗）以及卡介苗无应答的非肌层浸润性疾病（帕博利珠单抗）。靶向Nectin-4的恩诺单抗维德汀在铂类/免疫治疗后已证明有效。最重要的是，恩诺单抗维德汀联合帕博利珠单抗以前所未有的生存获益重新定义了一线治疗（中位总生存期31.5个月 vs 化疗的16.1个月；风险比0.47），而纳武利尤单抗联合吉西他滨-顺铂改善了适合顺铂治疗患者的结局。关键挑战包括管理独特的毒性特征、优化治疗顺序以及开发可靠的生物标志物。 **结论：** 联合疗法提供了最有前景的前进路径，未来研究需关注耐药机制、生物标志物开发以及将这些疗法扩展至更早期的疾病阶段。
肿瘤(癌症)患者之家