尽管脑转移瘤（BM）在非鳞状非小细胞肺癌患者（ns-NSCLC）的诊断中很常见，但它们在随机对照试验中大多被排除在外。本回顾性研究的目的是评估一线免疫检查点抑制剂（ICI）在这些患者中的实际疗效。本研究评估了首线ICI为基础的治疗与化疗（CT）在被诊断为BM且无可靶向改变的ns-NSCLC患者中的颅内和全身疗效，并进行比较。患者根据系统治疗分为CT组、ICI组和CT-ICI组。主要终点是总生存期（OS），使用Kaplan-Meier和Cox方法进行比较。次要终点是颅内无进展生存期（icPFS）。在2018年01月至2021年05月期间，纳入了118名患者（CT组52名、ICI组38名和CT-ICI组28名）。中位随访时间为30.0个月。CT组、ICI组和CT-ICI组的患者中分别有75.0%、68.4%和67.9%接受了颅内放疗（p=0.805）。校正后发现，ICI组和CT-ICI组与CT组相比，OS更好（HR=0.46，95%CI：0.23-0.89，和HR=0.52，95%CI：0.27-1.01，分别）。ICI组和CT-ICI组与CT组相比，颅内无进展的风险降低了54%（HR=0.46，95%CI：0.25-0.84）和59%（HR=0.41，95%CI：0.23-0.77）。立体定向放射外科与仅系统治疗相比，与颅内无进展生存期（icPFS）增加相关（HR=0.51，95%CI：0.29-0.92），而整脑放疗则未与其相关。真实生活中在诊断时患有BM的ns-NSCLC患者，一线使用ICI进行治疗与仅CT相比，显示出OS和icPFS的益处。应进行前瞻性评估系统治疗和局部治疗的理想类型和顺序。版权所有 © 2023 Elsevier B.V.。保留所有权利。

Although brain metastases (BM) at diagnosis are common in non-squamous NSCLC patients (ns-NSCLC), they have been mostly excluded from randomized trials. The aim of this retrospective study was to evaluate real-word outcomes of frontline immune checkpoint inhibitor (ICI) in these patients.Our study assess the intracranial and overall efficacy of first-line ICI-based therapy compared to chemotherapy (CT) in ns-NSCLC patients diagnosed with BM, showing no targetable alterations. Patients were divided according to systemic therapy: CT, ICI, or CT-ICI. Primary endpoint was overall survival (OS), compared using Kaplan-Meier and Cox methodology. Secondary endpoint was intracranial progression free survival (icPFS).Between 01 and 2018 and 05-2021, 118 patients were included (52 CT, 38 ICI and 28 CT-ICI). Median follow-up was 30.0 months. Intracranial radiotherapy was delivered for 75.0%, 68.4% and 67.9% of patients for CT, ICI and CT-ICI groups (p = 0.805). After adjustment, ICI and CT-ICI were associated with a better OS compared to CT (HR = 0.46, 95 %CI: 0.23-0.89, and HR = 0.52, 95 %CI: 0.27-1.01, respectively). ICI and CT-ICI were associated with a significant reduction in the risk of intracranial progression by 54% (HR = 0.46, 95 %CI: 0.25-0.84) and 59% (HR = 0.41, 95 %CI: 0.23-0.77) compared to CT. Stereotactic radiosurgery was associated with an increased icPFS compared to systemic therapy alone (HR = 0.51, 95% CI: 0.29 - 0.92), whereas whole-brain was not.Real-life ns-NSCLC patients with BM at diagnosis treated frontline with ICI presented OS and icPFS benefit compared to CT alone. A prospective assessment of the ideal type and sequence of systemic and local therapy should be conducted.Copyright © 2023 Elsevier B.V. All rights reserved.