Sintilimab Plus Axitinib for Advanced Fumarate Hydratase–Deficient Renal Cell Carcinoma

作者：Xingming Zhang, Haoyang Liu, Jiayu Liang, Junjie Zhao, Yongquan Wang, Yuntian Chen, Deying Kang, Qian Chen, Yaowen Zhang, Xiaoxue Yin, Yuhao Zeng, Zilin Wang, Xinan Sheng, Xin Yao, Kan Gong, Xiaodong Liu, Zhibin Chen, Mingxing Qiu, Wei Chen, Zongping Wang, Guangheng Luo, Tingting Zhou, Nanshan Yang, Xiuyi Pan, Ling Nie, Mengni Zhang, Junru Chen, Jinge Zhao, Xu Hu, Lijing Xu, Bo Tang, Jindong Dai, Haolin Liu, Yuchao Ni, Rui Huang, Qiang Wei, Xiang Li, Qiying He, Jiyan Liu, Pengfei Shen, Ni Chen, Zhenhua Liu, Guangxi Sun, Jin Yao, Hao Zeng · 发表于：JAMA Oncology · 年份：2025 · DOI：10.1001/jamaoncol.2025.2497 · 被引用次数：6 · 研究领域：Renal cell carcinoma treatment、Thyroid Cancer Diagnosis and Treatment、Renal and related cancers

Importance: Fumarate hydratase-deficient renal cell carcinoma (FH-deficient RCC) is a lethal kidney cancer that has limited therapeutic options. Objective: To evaluate the efficacy and safety of sintilimab plus axitinib for treatment of advanced FH-deficient RCC. Design, Setting, and Participants: This phase 2 multicenter, open-label, single-arm nonrandomized clinical trial enrolled patients with pathologically confirmed, treatment-naive, advanced FH-deficient RCC and an Eastern Cooperative Oncology Group Performance Status of 0 to 2 from July 1, 2021, to August 29, 2023, across 8 institutions in China. The data cutoff date was December 1, 2024. Intervention: Patients received sintilimab, 200 mg, intravenously every 3 weeks, combined with axitinib, 5 mg, orally twice daily, until disease progression, intolerable toxic effects, or withdrawal of consent. Main Outcomes and Measures: The primary end points were objective response rate (ORR) via Response Evaluation Criteria in Solid Tumors, version 1.1, and progression-free survival (PFS). Secondary end points included safety, overall survival, disease control rate (proportion of patients with complete or partial response or stable disease for ≥6 months), duration of response, and exploratory genomic-associated outcomes. Results: Of 52 patients screened for eligibility, 41 patients (median [range] age, 36 [18-75] years; 10 [24%] female) were enrolled. The median (range) duration of follow-up was 26.0 (0.7-41.6) months. Overall, OR...