1Department of Neurological Surgery, The Ohio State University Wexner Medical Center, Columbus, Ohio.
2The Ohio State University College of Medicine, Columbus, Ohio; and.
Neurosurg Focus. 2023 Aug;55(2):E2. doi: 10.3171/2023.5.FOCUS23122.
Immune checkpoint inhibitor (ICI) efficacy in the treatment of metastatic renal cell carcinoma (RCC) without brain metastases (BMs) is well established in several clinical trials; however, patients with BMs were typically excluded from these trials. Therefore, the efficacy of ICI in the treatment or prevention of BM remains unclear. The primary aim of the study was to address the efficacy of ICI in treatment of patients with RCC BMs compared with patients receiving targeted therapies. A secondary aim was to evaluate the risk of RCC BM development among patients who received ICI versus targeted therapies early in their treatment course.
A retrospective single-center review between 2011 and 2018 identified 425 patients treated for metastatic RCC. The study group included patients who received ICI and/or targeted therapies during their disease. Data analyzed included demographic information, systemic treatments, overall survival from RCC diagnosis (OSRCC) and from BM diagnosis (OSBM), and BM development. Fisher's exact test was used to evaluate the frequency of BM occurrence. Survival was assessed using Kaplan-Meier curves and log-rank tests.
Of the 425 patients, 125 received ICI and 300 were treated with molecular targeted agents only during their clinical course. BMs occurred in 113 (9.5%) of the 425 patients. Among patients with BMs, OSRCC was improved with the use of ICI (77.2 vs 25.2 months, p < 0.001), with 1-, 2-, and 5-year survival rates of 93.9%, 81.8%, and 62.6%, respectively. The use of ICI was associated with increased OSBM (21.7 vs 8.9 months, p = 0.001). The rate of BM development was lower when patients were treated with ICI (8/100 [8.0%]) compared with targeted therapy (47/267 [17.6%]) (OR 0.41, 95% CI 0.18-0.89; p = 0.021).
ICI was associated with improved OSRCC and OSBM in patients with BMs and decreased the probability of BM development in patients with metastatic RCC. Prospective trials are needed to further evaluate optimal use of ICI in treatment of RCC BMs.
多项临床试验已证实免疫检查点抑制剂（ICI）在治疗无脑转移（BM）的转移性肾细胞癌（RCC）方面具有显著疗效；然而，这些试验通常排除了有 BM 的患者。因此，ICI 治疗或预防 BM 的疗效仍不明确。本研究的主要目的是评估与接受靶向治疗的患者相比，ICI 在治疗 RCC BM 患者中的疗效。次要目的是评估在治疗过程中早期接受 ICI 与靶向治疗的患者中 RCC BM 发展的风险。
回顾性分析了 2011 年至 2018 年间的单中心资料，共纳入 425 例转移性 RCC 患者。研究组包括接受 ICI 和（或）靶向治疗的患者。分析的数据包括人口统计学信息、全身治疗、从 RCC 诊断（OSRCC）和从 BM 诊断（OSBM）的总生存时间，以及 BM 的发生情况。采用 Fisher 确切检验评估 BM 发生的频率。采用 Kaplan-Meier 曲线和对数秩检验评估生存情况。
425 例患者中，125 例接受 ICI 治疗，300 例仅接受分子靶向药物治疗。425 例患者中，有 113 例（9.5%）发生 BM。在发生 BM 的患者中，ICI 的使用改善了 OSRCC（77.2 个月 vs 25.2 个月，p<0.001），1、2、5 年生存率分别为 93.9%、81.8%和 62.6%。ICI 的使用与 OSBM 的延长相关（21.7 个月 vs 8.9 个月，p=0.001）。与靶向治疗相比，ICI 治疗患者的 BM 发生率较低（8/100[8.0%]比 47/267[17.6%]）（OR 0.41，95%CI 0.18-0.89；p=0.021）。
ICI 可改善有 BM 的患者的 OSRCC 和 OSBM，并降低转移性 RCC 患者的 BM 发生概率。需要前瞻性试验进一步评估 ICI 在治疗 RCC BM 中的最佳应用。
