Neoadjuvant combined chemotherapy and immunotherapy for upper tract urothelial carcinoma: Preliminary results from a phase II study.

作者：Xingang Bi, Wen Zhang, Yaoyu Xie, Jie Wu, Honglei Cui, Chuanzhen Cao, Lu Li, Li Wen, Youyan Guan, Hongzhe Shi, Zhendong Xiao, Zhichao Jiang, Yongkun Sun, Shan Zheng, J. Zhang, Aiping Zhou, Jianzhong Shou · 发表于：Journal of Clinical Oncology · 年份：2024 · DOI：10.1200/jco.2024.42.4_suppl.636 · 被引用次数：1 · 研究领域：Bladder and Urothelial Cancer Treatments、Urinary and Genital Oncology Studies、Renal cell carcinoma treatment

636 Background: Upper Tract Urothelial Carcinoma (UTUC) presents a challenging prognosis even after Radical Nephroureterectomy (RNU), and postoperative renal insufficiency further limits the options for adjuvant therapy. The efficacy of neoadjuvant chemotherapy for UTUC remains uncertain as past studies have not shown satisfactory results and have mostly been retrospective. There is an urgent need for a more promising regimen. This phase II study aims to investigate the efficacy and safety of a combination of chemotherapy (Gemcitabine/Cisplatin) and PD-1 inhibitor (Toripalimab) as neoadjuvant treatment (NT) in UTUC patients. Methods: We planned to enroll 34 UTUC patients with either cT1N0M0 (high grade) or cT2-3N0M0, confirmed by ureterorenoscopy biopsy and imaging. The treatment regimen included three or four cycles of NT (Gemcitabine, 800mg/m 2 , days 1 and 8/Cisplatin, 60mg/m 2 , day 1/Toripalimab, 240mg, day 1 of a 21-day cycle), followed by RNU and pelvic lymphadenectomy. The primary outcome was the pathological complete response (pCR) rate, with secondary outcomes including significant downstaging (≤pT1) rate, disease control rate (DCR), and safety. Results: To date, 17 patients have been accrued since August 1st, 2020, and recruitment is ongoing. Fifteen patients have completed treatments and were preliminarily analyzed, with two patients still undergoing treatment. The median age was 66.0 years, with 53.3% being male. The majority of patients had unifocal tumors, with...