Background/objectives: The real-world survival of patients with unresectable hepatocellular carcinoma (uHCC) treated with lenvatinib has been explored retrospectively with a small sample size. We conducted a prospective observational 2-year extension study (510 study) of a 1-year observational post-marketing study of lenvatinib (504 study) to evaluate the long-term overall survival (OS) of patients with uHCC treated with lenvatinib and associated factors with a large sample size. Methods: Patients with uHCC included (July 2018 to January 2019) in the 504 study and who consented were eligible for the 510 study and were followed for up to 3 years after lenvatinib treatment initiation. Using the data from the 504 study and 510 study of the 504 study analysis set, we estimated the OS, the time from the first lenvatinib dose to all-cause death by the Kaplan–Meier method (ClinicalTrials.Gov Registration ID, 504 study: NCT03663114; 510 study: NCT04008082). Results: The 703 patients included in the analysis were followed for a median period (min, max) of 12.5 months (0.1, 44.8). The median OS (95% confidence interval) was 16.6 months (15.4, 18.5). OS was significantly (p< 0.05) associated with bile duct invasion (hazard ratio [HR]: 1.621), portal vein invasion (HR: 1.365), ≥ 4 intrahepatic lesions (HR: 1.437), extrahepatic lesions (HR: 1.357), Child–Pugh B/C (HR: 1.515), mALBI Grade 2a (HR: 1.331), and Grade ≥ 2b (HR: 1.811). Conclusions: This large-scale, prospective, real-world study demonstrated a long OS, comparable to that reported in the global Phase III REFLECT trial. More advanced-stage tumors and worse hepatic function have been suggested as OS-associated factors, consistent with previous reports.
背景/目的：既往小样本回顾性研究探讨了乐伐替尼治疗不可切除肝细胞癌（uHCC）患者的真实世界生存情况。我们在乐伐替尼为期一年的上市后观察性研究（504研究）基础上，开展了一项为期两年的前瞻性观察性扩展研究（510研究），旨在通过大样本量评估乐伐替尼治疗uHCC患者的长期总生存期（OS）及其相关因素。方法：纳入504研究（2018年7月至2019年1月）中同意参与的uHCC患者进入510研究，自乐伐替尼治疗开始随访最长3年。基于504研究分析集及其510扩展研究的数据，采用Kaplan-Meier法评估OS（定义为从首次使用乐伐替尼至全因死亡的时间）（临床试验注册号：504研究：NCT03663114；510研究：NCT04008082）。结果：纳入分析的703例患者中位随访时间（最小值，最大值）为12.5个月（0.1，44.8）。中位OS（95%置信区间）为16.6个月（15.4，18.5）。OS与以下因素显著相关（p<0.05）：胆管侵犯（风险比[HR]：1.621）、门静脉侵犯（HR：1.365）、≥4个肝内病灶（HR：1.437）、肝外病灶（HR：1.357）、Child-Pugh B/C级（HR：1.515）、mALBI分级2a级（HR：1.331）及≥2b级（HR：1.811）。结论：这项大规模前瞻性真实世界研究证实了乐伐替尼治疗uHCC患者可获得较长的OS，与全球III期REFLECT试验报告结果相当。更晚期的肿瘤分期和更差的肝功能被证实是OS的相关因素，这与既往报道一致。
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