Department of Oncology, Odense University Hospital, Odense, Denmark.
Department of Clinical Research, University of Southern Denmark, Odense, Denmark.
Acta Oncol. 2023 Aug;62(8):861-870. doi: 10.1080/0284186X.2023.2238544. Epub 2023 Aug 3.
Convincing results from randomized controlled trials (RCTs) have led to increasing use of immune checkpoint inhibitors (ICI) as part of standard therapies in real-world (RW) scenarios. However, RW patients differ clinically from RCT populations and might have reduced long-term survival. Currently, only sparse data on 3-5-year survival rate for RW patients with advanced non-small cell lung cancer (NSCLC) treated with ICI exist.
A multicenter study was performed including 729 patients with advanced NSCLC receiving monotherapy with ICI (retrospective data ( = 566) and prospective data ( = 163)). Detailed baseline clinical characteristics, programmed death-ligand 1 (PD-L1) tumor proportion score (TPS), and baseline haematological count were registered. Kaplan-Meier estimates and log-rank test were used for survival analyses, Cox regression for determination of prognostic factors.
Median time of follow-up (FU) was 48.7 months (IQR 37.2-54.3). Median overall survival (OS) in first line treatment was 20.4 months (IQR 8.5-45.0) compared to 11.4 months (IQR 4.6-27.1) in ≥2nd line (HR 1.48, 95% CI 1.25-1.75). Estimated probability of OS was 30% at 3 years, 23% at 4 years, and 13% at 5 years in first line compared to 17, 13, and 11% in ≥2nd line, respectively. For those with performance status (PS) 2, the 2-year OS rate was 32% (95% CI 0.22-0.43) compared to 5% (95% CI 0.01-0.15) in patients with PD-L1 ≥ 50% versus <50%, respectively.
Compared to RCTs, long-term OS and PFS rates are lower in real-world patients treated with ICI in first line but much improved compared to historic rates on chemotherapy. A promising flattening of both the OS and progression free survival curves illustrates that also a subset of real-world patients obtain long-term remission. Patients with PS 2 and PD-L1 ≥ 50% may obtain clinically meaningful 2-year PFS and OS rates.
随机对照试验（RCT）的令人信服的结果导致免疫检查点抑制剂（ICI）越来越多地被用作真实世界（RW）环境中标准治疗的一部分。然而，RW 患者在临床上与 RCT 人群不同，可能长期生存率降低。目前，关于接受 ICI 治疗的晚期非小细胞肺癌（NSCLC）RW 患者 3-5 年生存率的稀疏数据仅存在。
进行了一项多中心研究，纳入 729 名接受 ICI 单药治疗的晚期 NSCLC 患者（回顾性数据（ = 566）和前瞻性数据（ = 163））。详细登记了基线临床特征、程序性死亡配体 1（PD-L1）肿瘤比例评分（TPS）和基线血液学计数。使用 Kaplan-Meier 估计和对数秩检验进行生存分析，使用 Cox 回归确定预后因素。
中位随访（FU）时间为 48.7 个月（IQR 37.2-54.3）。一线治疗的中位总生存期（OS）为 20.4 个月（IQR 8.5-45.0），而≥2 线治疗的中位 OS 为 11.4 个月（IQR 4.6-27.1）（HR 1.48，95%CI 1.25-1.75）。在一线治疗中，估计的 3 年 OS 概率为 30%，4 年 OS 概率为 23%，5 年 OS 概率为 13%，而≥2 线治疗的相应概率分别为 17%、13%和 11%。对于 PS 2 的患者，2 年 OS 率为 32%（95%CI 0.22-0.43），而 PD-L1≥50%的患者为 5%（95%CI 0.01-0.15），分别。
与 RCT 相比，接受 ICI 一线治疗的 RW 患者的长期 OS 和 PFS 率较低，但与化疗的历史数据相比有很大改善。OS 和无进展生存期曲线的显著平坦化表明，也有一部分 RW 患者获得了长期缓解。PS 2 和 PD-L1≥50%的患者可能获得具有临床意义的 2 年 PFS 和 OS 率。
