Pd20-11 Neoadjuvant Toripalimab and Chemotherapy for Locally Advanced Upper Urinary Tract Carcinoma: A Pilot Study for a Phase Ii Trial

You have accessJournal of UrologyBladder Cancer: Upper Tract Transitional Cell Carcinoma I (PD20)1 May 2024PD20-11 NEOADJUVANT TORIPALIMAB AND CHEMOTHERAPY FOR LOCALLY ADVANCED UPPER URINARY TRACT CARCINOMA: A PILOT STUDY FOR A PHASE II TRIAL Xinyang Liao, Yige Bao, and Qiang Wei Xinyang LiaoXinyang Liao , Yige BaoYige Bao , and Qiang WeiQiang Wei View All Author Informationhttps://doi.org/10.1097/01.JU.0001009556.54476.eb.11AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Upper urinary tract urothelial carcinomas (UTUC) are commonly invasive when initially diagnosed. The pathological complete response (pCR) rate of neoadjuvant chemotherapy for locally advanced UTUC is 10-20%. No evidence proved the effectiveness and safety of neoadjuvant immunotherapy combined with chemotherapy for locally advanced UTUC. Toripalimab is an innovative anti-PD-1 monoclonal antibody approved in China across multiple cancer indications. In this pilot study, we aimed to explore the efficacy and safety profile of neoadjuvant toripalimab combined with chemotherapy in locally advanced UTUC. Based on these data, a prospective phase II study of neoadjuvant therapy for UTUC is being conducted. METHODS: Twenty-one patients with locally advanced UTUC were included. Patients received four cycles of toripalimab (240 mg p.o.) plus gemcitabine (1000 mg/m2 on D1 and D8) and cisplatin (75 mg/m2, D1) every three weeks for 3-4 cycles before radical nephroureterectomy (RU). Patients were followed up monthly by clinic appointments until September 30, 2023. Adverse events were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. The primary outcomes included pCR rate, objective response rate (ORR), and serious adverse event (SAE) rate. The secondary outcomes included disease control rate (DCR), progression-free survival (PFS), and grade 3 or worse treatment-emergent adverse event rate. RESULTS: Among the 21 included patients, two patients were lost to follow-up, and one patient refused to receive radical nephroureterectomy when considered a clinical complete response. Of the remaining 18 patients, the median age was 67.5 (range: 45-79). Fifteen (83.33%) patients were male. The median follow-up was 66 weeks (range: 27-83 weeks). Six patients (33.33%) achieved pCR. Eight patients (44.44%) achieved pathological partial response. Three patients (16.67%) had stable disease. One patient (5.56%) acquired progressive disease. The ORR was 77.78% (14/18). The DCR was 94.4% (17/18). The median PFS was not reached since all patients had survived till the endpoint. No SAE occurred. Seven patients (38.89%) had grade 3 or worse treatment-emergent adverse events during chemotherapy cycles. This study served as a pilot study for a prospective, single-arm phase II trial (WUTSUP-01) of neoadjuvant toripalimab and chemotherapy for locally advanced UTUC (cT2-4N0-2M0). CONCLUSIONS: Neoadjuvant toripalimab combined with chemotherapy demonstrated promising efficacy with a 33.33% pCR rate and a 77.78% ORR in locally advanced UTUC. The combined therapy also showed a manageable safety profile. The ongoing WUTSUP-01 trial will further evaluate the efficacy and safety of toripalimab with chemotherapy in UTUC patients. Source of Funding: This work was not supported by any funding © 2024 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 211Issue 5SMay 2024Page: e451 Advertisement Copyright & Permissions© 2024 by American Urological Association Education and Research, Inc.Metrics Author Information Xinyang Liao More articles by this author Yige Bao More articles by this author Qiang Wei More articles by this author Expand All Advertisement PDF downloadLoading ...