Oh Jong Jin, Hong Sung Kyu
Department of Urology, Seoul National University Bundang Hospital, Seongnam, Korea.
Department of Urology, Seoul National University College of Medicine, Seoul, Korea.
Investig Clin Urol. 2025 Sep;66(5):375-382. doi: 10.4111/icu.20250008.
Urothelial carcinoma, the most common malignancy of the urinary tract, presents a significant challenge, particularly in its metastatic stage, where prognosis remains poor despite advancements in treatment. Historically, platinum-based chemotherapy has been the standard first-line therapy, achieving moderate response rates but limited long-term survival. Recent breakthroughs have introduced immune checkpoint inhibitors, antibody-drug conjugates (ADCs), and targeted therapies as more effective alternatives. Enfortumab vedotin plus pembrolizumab has demonstrated superior efficacy as a first-line treatment, improving overall survival (OS) and objective response rates compared to chemotherapy. Maintenance therapy with avelumab has further prolonged survival in patients responding to initial platinum-based chemotherapy. Additionally, sacituzumab govitecan, an ADC targeting Trop-2, and erdafitinib, a fibroblast growth factor receptor (FGFR) inhibitor, have provided promising options for patients with refractory disease or FGFR alterations. The evolving treatment paradigm now prioritizes biomarker-driven, personalized approaches over traditional chemotherapy-based regimens. However, challenges remain in optimizing treatment sequencing and managing toxicity. Future research should focus on refining patient selection criteria and exploring novel combination therapies to enhance efficacy and durability of response.
尿路上皮癌是泌尿系统最常见的恶性肿瘤，带来了重大挑战，尤其是在转移阶段，尽管治疗取得了进展，但预后仍然很差。从历史上看，铂类化疗一直是标准的一线治疗方法，缓解率中等，但长期生存率有限。最近的突破引入了免疫检查点抑制剂、抗体药物偶联物（ADC）和靶向治疗作为更有效的替代方案。与化疗相比，恩杂鲁胺联合帕博利珠单抗作为一线治疗已显示出卓越疗效，提高了总生存期（OS）和客观缓解率。阿维鲁单抗维持治疗进一步延长了对初始铂类化疗有反应的患者的生存期。此外，靶向Trop-2的ADC药物戈沙妥珠单抗和成纤维细胞生长因子受体（FGFR）抑制剂厄达替尼，为难治性疾病或FGFR改变的患者提供了有希望的选择。不断演变的治疗模式现在优先考虑基于生物标志物的个性化方法，而不是传统的基于化疗的方案。然而，在优化治疗顺序和管理毒性方面仍然存在挑战。未来的研究应专注于完善患者选择标准，并探索新的联合治疗方法，以提高疗效和反应的持久性。
