将lenvatinib与程序性细胞死亡蛋白-1（PD-1）抑制剂结合用于治疗不可切除的肝细胞癌（uHCC）已得到研究。本研究旨在研究亚洲大量uHCC患者中lenvatinib加PD-1抑制剂治疗的现实有效性和与生存相关的预后因素，即使全球LEAP-002研究未能实现主要终点。选择接受lenvatinib和PD-1抑制剂治疗的uHCC患者，主要终点为总生存期（OS）和无进展生存期（PFS），次要终点为客观缓解率（ORR）和不良事件（AE）。还分析了影响生存的预后因素。总共回顾性评估了来自中国两个医疗中心的378名uHCC患者。患者的中位年龄为55岁，男性占86.5％。乙型肝炎病毒（HBV）感染（89.9％）是uHCC的主要病因。中位OS为17.8（95％置信区间（CI）14.0-21.6）个月，中位PFS为6.9（95％CI 6.0-7.9）个月，最佳ORR和疾病控制率（DCR）分别为19.6％和73.5％。在多元分析中，Child-Pugh分级，巴塞罗那肝癌诊所分期，东方合作组织肿瘤学小组表现状态评分，涉及器官，肿瘤负担分数以及与局部治疗的联合是OS的独立预后因素。总共100％和57.9％的患者经历了所有级别和3/4级别的治疗相关AE。这项lenvatinib加PD-1抑制剂治疗的现实研究证明，在中国的uHCC患者中具有长期生存和相当的ORR和DCR。联合疗法的耐受性是可接受的，但必须严密监测。 © 2023作者（们）。

Combining lenvatinib with a programmed cell death protein-1 (PD-1) inhibitor has been explored for the treatment of un-resectable hepatocellular carcinoma (uHCC). This study aimed to investigate the real-world efficacy of and prognostic factors for survival associated with lenvatinib plus PD-1 inhibitor treatment in a large cohort of Asian uHCC patients even the global LEAP-002 study failed to achieve the primary endpoints.Patients with uHCC treated with lenvatinib and PD-1 inhibitors were included. The primary endpoints were overall survival (OS) and progression-free survival (PFS), and the secondary endpoints were the objective response rate (ORR) and adverse events (AEs). Prognostic factors for survival were also analyzed.A total of 378 uHCC patients from two medical centers in China were assessed retrospectively. The median patient age was 55 years, and 86.5% of patients were male. Hepatitis B virus (HBV) infection (89.9%) was the dominant etiology of uHCC. The median OS was 17.8 (95% confidence interval (CI) 14.0-21.6) months. The median PFS was 6.9 (95% CI 6.0-7.9) months. The best ORR and disease control rate (DCR) were 19.6% and 73.5%, respectively. In multivariate analysis, Child‒Pugh grade, Barcelona Clinic Liver Cancer stage, Eastern Cooperative Oncology Group performance status score, involved organs, tumor burden score, and combination with local therapy were independent prognostic factors for OS. A total of 100% and 57.9% of patients experienced all-grade and grade 3/4 treatment-emergent AEs, respectively.This real-world study of lenvatinib plus PD-1 inhibitor treatment demonstrated long survival and considerable ORRs and DCRs in uHCC patients in China. The tolerability of combination therapy was acceptable but must be monitored closely.© 2023. The Author(s).