在多个临床试验中，注射免疫检查点抑制剂（ICI）用于治疗无脑转移的转移性肾细胞癌（RCC）的疗效已得以确立。然而，这些试验通常将患有脑转移的患者排除在外，因此ICI在治疗或预防脑转移的疗效仍不明确。本研究的主要目的是比较ICI在治疗患有RCC脑转移的患者中的疗效与接受靶向治疗的患者；次要目的是评估接受ICI与靶向治疗早期治疗的患者中发展RCC脑转移的风险。本研究是一项回顾性单中心研究，时间跨度为2011年至2018年，共纳入425名转移性RCC患者。研究组包括在疾病过程中接受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、自RCC诊断以来的总体生存率（OSRCC）和自脑转移诊断以来的总体生存率（OSBM），以及脑转移发生情况。使用Fisher确切概率检验评估脑转移发生的频率。使用Kaplan-Meier曲线和log-rank检验评估生存情况。在425名患者中，125例接受了ICI治疗，而300例在其临床过程中仅接受了分子靶向药物治疗。在这425名患者中，113例（9.5%）发生了脑转移。在患有脑转移的患者中，ICI的使用改善了OSRCC（77.2 vs 25.2个月，p < 0.001），1、2和5年的生存率分别为93.9％、81.8％和62.6％。ICI的使用与OSBM的增加相关（21.7 vs 8.9个月，p = 0.001）。与靶向治疗相比，ICI治疗的患者脑转移的发生率较低（8/100 [8.0%] vs 47/267 [17.6%]）（OR：0.41，95％CI：0.18-0.89，p = 0.021）。ICI与患有脑转移的患者的OSRCC和OSBM改善以及转移性RCC患者出现脑转移的可能性减少有关。需要进一步进行前瞻性试验来评估ICI在治疗RCC脑转移中的最佳使用方法。

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.