Neoadjuvant treatment with disitamab vedotin plus perioperative toripalimab in patients with muscle-invasive bladder cancer (MIBC) with HER2 expression: Updated efficacy and safety results from the phase II RC48-C017 trial.

作者：Xinan Sheng, Cuijian Zhang, Yongpeng Ji, Li Zhou, Benkui Zou, Hang Huang, Yonghua Wang, Kaiwei Yang, Xue Bai, Dan Feng, Yong Yang, Jiasheng Bian, Zhixian Yu, Haitao Niu, Peng Du, Jianmin Fang, Zhisong He, Jun Guo · 发表于：Journal of Clinical Oncology · 年份：2025 · DOI：10.1200/jco.2025.43.5_suppl.665 · 被引用次数：18 · 研究领域：Bladder and Urothelial Cancer Treatments、Cancer Immunotherapy and Biomarkers、Cancer, Stress, Anesthesia, and Immune Response

665 Background: This single-arm phase II trial was conducted to evaluate the efficacy and safety of neoadjuvent disitamab vedotin (DV, a HER2-targetted monoclonal antibody conjugated with monomethyl auristatin E) plus perioperative toripalimab (an anti-PD-1 inhibitor) in MIBC patients (pts) with HER2 expression. The preliminary results showed promising efficacy and acceptable safety with neoadjuvant treatment with DV plus toripalimab in pts with HER2-expressing MIBC (Sheng, et al. ASCO Annual meeting 2024). Methods: Key eligibility criteria included previously untreated MIBC (cT2-4aN0-1M0) with HER2 expression (immunohistochemistry [IHC] ≥1+ by local test), and eligible for curative-intent radical cystectomy and pelvic lymph node dissection (RC+PLND). Pts received DV (2 mg/kg) plus toripalimab (3 mg/kg) every 2 weeks for 6 cycles at neoadjuvant phase. After RC+PLND, pts received adjuvant toripalimab (3 mg/kg every 2 weeks) for up to 20 cycles. The primary endpoint was pathological complete response (pCR, ypT0N0) rate assessed by the investigators; secondary endpoints included pathological response rate (≤ypT1N0M0), overall survival, safety, etc. Here we present the updated efficacy and safety results and post-hoc event-free survival (EFS) analysis with data cutoff date (DCO) of September, 2024. Results: As of DCO, patient enrollment was completed with 47 pts enrolled and treated (including 10.6% pts with HER2 IHC 1+, 57.4% IHC 2+, and 31.9% IHC 3+; 83.0% pts at baseline T2-4N...