Gottlieb Josh, Linehan Jennifer, Murray Katie S
Department of Urologic Oncology, St. John's Cancer Institute, Santa Monica, CA, USA.
Department of Urology, NYU Langone Medical Center, New York, NY, USA.
Transl Androl Urol. 2023 Sep 30;12(9):1449-1455. doi: 10.21037/tau-23-69. Epub 2023 Aug 1.
Localized upper tract urothelial carcinoma (UTUC) is a difficult disease for clinicians to treat, due to the multitude of oncological and patient factors to consider. Despite the challenges of diagnostic staging, endoscopic management, and disease recurrence, there is still a need for local therapeutic options that do not subject patients to the morbidities of radical nephroureterectomy (RNU). Intraluminal chemotherapies have allowed for improved oncological control in patients with low-grade disease receiving renal-sparing treatment approaches. This narrative review discusses the treatment modalities available for localized low-grade UTUC, with a focus on the current status of chemoablation. The OLYMPUS trial was a pivotal study that lead to the Food and Drug Administration (FDA) approval of UGN-101 (mitomycin-C) in April 2020 for the treatment of low-grade UTUC, and intraluminal chemotherapy is now a widely used modality for managing this disease. The trial reported a complete response (CR) rate of 59%, and an estimated treatment durability of 82% at 1 year. However, a concern was the reported 44% ureteral stricture rate using the retrograde approach. More research is currently underway to determine the ideal instillation method for intraluminal therapies (e.g., retrograde vs. antegrade). Lastly, we discuss upcoming treatment options. Newer novel agents like padeliporfin vascular targeted photodynamic (VTP) therapy (brand name TOOKAD) are currently being studied, which will in hope provide additional treatment options for UTUC patients.
局限性上尿路尿路上皮癌（UTUC）对临床医生来说是一种难以治疗的疾病，因为需要考虑众多肿瘤学和患者因素。尽管在诊断分期、内镜治疗和疾病复发方面存在挑战，但仍需要局部治疗方案，使患者无需承受根治性肾输尿管切除术（RNU）带来的并发症。腔内化疗已使接受保留肾治疗方法的低级别疾病患者的肿瘤控制得到改善。这篇叙述性综述讨论了局限性低级别UTUC可用的治疗方式，重点是化学消融的现状。OLYMPUS试验是一项关键研究，该研究于2020年4月使美国食品药品监督管理局（FDA）批准UGN-101（丝裂霉素-C）用于治疗低级别UTUC，腔内化疗目前已成为治疗该疾病广泛使用的方式。该试验报告的完全缓解（CR）率为59%，1年时估计的治疗持续率为82%。然而，一个问题是报告的逆行给药法输尿管狭窄率为44%。目前正在进行更多研究以确定腔内治疗的理想灌注方法（例如，逆行与顺行）。最后，我们讨论即将出现的治疗选择。目前正在研究像帕德立卟啉血管靶向光动力（VTP）疗法（商品名TOOKAD）这样更新的新型药物，有望为UTUC患者提供更多治疗选择。
