非透明细胞肾细胞癌 (nccRCC) 患者的治疗选择有限。在卡博替尼加纳武单抗的 2 期试验中，nccRCC 患者获得了良好的客观缓解率 (ORR)。我们现在报告乳头状、未分类或易位相关肾细胞癌患者中位随访 34 个月的最新疗效和安全性结果。卡博替尼和纳武单抗以标准剂量给予经过零线或一线全身治疗后进展的转移性 nccRCC 患者。主要终点是根据实体瘤 v1.1 中的反应评估标准的 ORR。次要终点包括无进展生存期（PFS）、总生存期（OS）和不良事件。四十名患者接受治疗。幸存者中位随访 34 个月时，ORR 为 48%（95% 置信区间 [CI] 31.5-63.9%）。中位 PFS 为 13 个月 (95% CI 7-16)； 12 个月和 24 个月的 PFS 率分别为 51% (95% CI 34-65%) 和 23% (95% CI 11-37%)。中位 OS 为 28 个月 (95% CI 23-43)； 18 个月和 36 个月 OS 率分别为 70% (95% CI 53-82%) 和 44% (95% CI 28-60%)。卡博替尼和纳武单抗没有发现新的安全信号。这项扩展的随访分析证明了有希望的疗效，并强调了卡博替尼加纳武单抗在转移性 nccRCC 患者中持续缓解的潜力。患者摘要：我们评估了接受卡博替尼纳武单抗治疗的非透明细胞 (NCC) 型转移性肾癌患者的结局。我们发现 48% 的患者对治疗有反应，并且没有出现意外的副作用。在对治疗有反应的患者中，反应持续时间中位数为 17 个月。我们的结论是，卡博替尼纳武单抗是一种安全有效的 NCC 肾癌治疗方法。版权所有 © 2024。由 Elsevier B.V. 出版。

Treatment options are limited for patients with non-clear cell renal cell carcinoma (nccRCC). Patients with nccRCC experienced a favorable objective response rate (ORR) in a phase 2 trial of cabozantinib plus nivolumab. We now report updated efficacy and safety results at median follow-up of 34 mo for patients with papillary, unclassified, or translocation-associated RCC. Cabozantinib and nivolumab were administered at standard doses to patients with metastatic nccRCC that had progressed on zero or one line of systemic therapy. The primary endpoint was the ORR according to Response Evaluation Criteria in Solid Tumors v1.1. Secondary endpoints included progression-free survival (PFS), overall survival (OS), and adverse events. Forty patients were treated. At median follow-up of 34 mo for survivors, the ORR was 48% (95% confidence interval [CI] 31.5-63.9%). Median PFS was 13 mo (95% CI 7-16); the 12-mo and 24-mo PFS rates were 51% (95% CI 34-65%) and 23% (95% CI 11-37%), respectively. Median OS was 28 mo (95% CI 23-43); the 18-mo and 36-mo OS rates were 70% (95% CI 53-82%) and 44% (95% CI 28-60%), respectively. No new safety signals were seen with cabozantinib and nivolumab. This extended follow-up analysis demonstrates promising efficacy, and highlights the potential for sustained responses with cabozantinib plus nivolumab in patients with metastatic nccRCC. PATIENT SUMMARY: We evaluated outcomes for patients with metastatic kidney cancer of the non-clear cell (NCC) type who were treated with cabozantinib + nivolumab. We found that 48% of the patients responded to the treatment, and there were no unexpected side effects. Among patients who responded to the treatment, the response lasted for a median of 17 months. We conclude that cabozantinib + nivolumab is a safe and effective treatment for NCC kidney cancer.Copyright © 2024. Published by Elsevier B.V.