文章：

PD-L1表达≥50%的转移性非小细胞肺癌一线帕博利珠单抗单药治疗的真实世界安全性与疗效：一项意大利全国多中心队列研究（“PEMBROREAL”研究）

Real-World Safety and Outcome of First-Line Pembrolizumab Monotherapy for Metastatic NSCLC with PDL-1 Expression ≥ 50%: A National Italian Multicentric Cohort (“PEMBROREAL” Study)

原文发布日期：8 May 2024

DOI: 10.3390/cancers16101802

类型: Article

开放获取: 是

英文摘要：

Results from the phase IIIKeynote-024clinical trial established pembrolizumab monotherapy as the first-line standard of care for patients with metastatic NSCLC who have PD-L1 expression ≥ 50%,EGFR, andALKwild-type tumors. However, given the differences between patients treated in routine clinical practice and those treated in a clinical trial, real-world data are needed to confirm the treatment benefit in standard practice. Given the lack of data on large cohorts of patients with long follow-ups, we designed an observational retrospective study of patients with metastatic NSCLC who were treated with pembrolizumab, starting from its reimbursement eligibility until December 2020. The primary endpoints were PFS and OS, determined using the Kaplan–Meier method. Response and safety were also evaluated. We followed 880 patients (median follow-up: 35.1 months) until February 2022. Median PFS and OS were 8.6 months (95% CI: 7.6–10.0) and 25.5 months (95% CI: 21.8–31.6), respectively. We also found that ECOG PS, PD-L1 expression, and habitual smoking were prognostic factors for PFS, while age, sex, ECOG PS, habitual smoking and histology had an impact on OS. Multivariable analysis confirms the prognostic role of PD-L1 for PFS and of ECOG for both PFS and OS. 39.9% of patients reported an adverse event, but only 6.3% of patients discontinued therapy due to toxicity. Our results suggest a long-term benefit of pembrolizumab in the first-line setting, as well as a safety profile consistent with the results ofKeynote-024. Many collected variables appear to influence clinical outcome, but results from these exploratory unadjusted analyses should be interpreted with caution.

摘要翻译： 

III期Keynote-024临床试验结果确立了帕博利珠单抗单药疗法作为PD-L1表达≥50%、EGFR及ALK野生型转移性非小细胞肺癌患者的一线标准治疗方案。然而，鉴于常规临床实践中的患者与临床试验患者存在差异，需要真实世界数据来验证标准实践中的治疗获益。由于缺乏长期随访的大规模患者队列数据，我们开展了一项观察性回顾研究，纳入从符合医保报销条件至2020年12月期间接受帕博利珠单抗治疗的转移性非小细胞肺癌患者。主要终点为采用Kaplan-Meier法评估的无进展生存期和总生存期，同时评估治疗反应及安全性。截至2022年2月，共随访880例患者（中位随访时间35.1个月）。中位无进展生存期和总生存期分别为8.6个月（95% CI：7.6-10.0）和25.5个月（95% CI：21.8-31.6）。研究还发现ECOG PS评分、PD-L1表达水平和长期吸烟史是无进展生存期的预后因素，而年龄、性别、ECOG PS评分、长期吸烟史和组织学类型对总生存期具有影响。多变量分析证实PD-L1对无进展生存期、ECOG PS对无进展生存期和总生存期均具有预后价值。39.9%的患者报告不良事件，但仅6.3%患者因毒性反应终止治疗。本研究结果表明帕博利珠单抗在一线治疗中具有长期获益，其安全性与Keynote-024研究结果一致。虽然多个收集变量可能影响临床结局，但需谨慎解读这些未经校正的探索性分析结果。

原文链接：

Real-World Safety and Outcome of First-Line Pembrolizumab Monotherapy for Metastatic NSCLC with PDL-1 Expression ≥ 50%: A National Italian Multicentric Cohort (“PEMBROREAL” Study)