Department of Urology, La Croix du Sud Hospital.
Department of Urology, Institut Universitaire du Cancer Toulouse - Oncopôle, Toulouse, France.
Curr Opin Urol. 2022 Sep 1;32(5):500-503. doi: 10.1097/MOU.0000000000001012. Epub 2022 Jul 15.
To evaluate intermediate clinical endpoints that have been proposed as potential surrogates for overall survival amongst patients with locally advanced and metastatic urothelial carcinoma.
Several endpoints have been proposed as potential surrogates for overall survival. They are: pathologic downstaging or complete response after neoadjuvant treatments and progression-free survival in the adjuvant setting and metastatic setting. Formal surrogacy, as per Prentice, has not been established among any of the aforementioned intermediate clinical endpoints and overall survival. Despite that, regulatory agencies have recently approved adjuvant nivolumab for patients with high-risk muscle invasive bladder cancer, based on the results of a trial that had disease-free survival as primary endpoint.
Despite the lack of proven surrogacy between progression-free survival and overall survival, this endpoint seems adequate for trial design and medication approval, as the recent case of adjuvant nivolumab demonstrates.
评估局部晚期和转移性尿路上皮癌患者的总生存期的潜在替代的中间临床终点。
已经提出了几种终点作为总生存期的潜在替代指标。它们是：新辅助治疗后的病理降期或完全缓解以及辅助治疗和转移性治疗中的无进展生存期。根据上述中间临床终点和总生存期，没有任何一个符合普伦蒂斯的正式替代指标。尽管如此，监管机构最近还是根据一项以无病生存期为主要终点的试验结果，批准了辅助用纳武单抗用于高危肌层浸润性膀胱癌患者。
尽管无进展生存期和总生存期之间缺乏已证实的替代关系，但正如辅助纳武单抗的最新案例所示，该终点似乎足以用于试验设计和药物批准。
