National Cancer Registry (RON), Portuguese Institute of Oncology of Lisbon Francisco Gentil (IPOLFG), Lisbon, Portugal.
Statistics & Epidemiology, National School of Public Health, Lisbon, Portugal.
Pharmacoepidemiol Drug Saf. 2020 Oct;29(10):1295-1302. doi: 10.1002/pds.5091. Epub 2020 Aug 25.
Immunotherapy is promising for lung cancer treatment, although at significant financial impact. The aim of this study was to evaluate the effectiveness and the efficacy-effectiveness gap of pembrolizumab in previously treated non-small cell lung cancer (NSCLC).
A population-based ambispective cohort study was conducted. Cases of interest were identified through the National Cancer Registry database and additional data sources. Patients aged ≥18 years, diagnosed with NSCLC and exposed to pembrolizumab, between 23 June 2016 and 31 October 2018, as second or later lines of treatment for advanced disease were included. Patients were followed-up until death or cut-off date (30 April 2019). Primary outcome was overall survival (OS). Secondary outcomes were progression-free survival (PFS), event-free survival (EFS), and adverse events (AEs) leading to treatment discontinuation. The efficacy-effectiveness gap was evaluated comparing results with clinical trial data.
A total of 181 patients were included. Median age was 63 years (range 33-94); 74.6% were male. Median treatment duration was 5.6 months (interquartile range: 1.4-10.4) and, at cut-off date, treatment had been discontinued in 141 patients, mainly due to disease progression. Median OS was 13.0 months (95% confidence interval [CI] 9.3-15.9) and 1-year OS was 53.1% (95% CI 45.2%-60.3%). Median PFS was 5.6 months (95% CI 4.6-7.2), median EFS was 4.7 months (95% CI 3.2-6.0), and treatment was discontinued due to AE in 8.3% of cases (n = 15). The efficacy-effectiveness gap seems to favor pembrolizumab use in clinical practice.
Real-world data suggest the performance of pembrolizumab to reflect the clinical trial outcomes in previously treated NSCLC.
免疫疗法在肺癌治疗中具有广阔的前景，尽管其费用也相当高昂。本研究旨在评估帕博利珠单抗在先前治疗过的非小细胞肺癌（NSCLC）患者中的有效性和疗效-效果差距。
本研究为基于人群的前瞻性队列研究。通过国家癌症登记数据库和其他数据源，确定感兴趣的病例。纳入 2016 年 6 月 23 日至 2018 年 10 月 31 日期间，年龄≥18 岁、诊断为 NSCLC 并接受帕博利珠单抗治疗的患者。患者接受二线或二线以上治疗晚期疾病。随访直至死亡或截止日期（2019 年 4 月 30 日）。主要结局是总生存期（OS）。次要结局包括无进展生存期（PFS）、无事件生存期（EFS）和因治疗中止而发生的不良事件（AE）。通过比较临床试验数据来评估疗效-效果差距。
共纳入 181 例患者。中位年龄为 63 岁（范围 33-94 岁）；74.6%为男性。中位治疗持续时间为 5.6 个月（四分位距：1.4-10.4），截止日期时，141 例患者已停止治疗，主要原因是疾病进展。中位 OS 为 13.0 个月（95%置信区间 [CI] 9.3-15.9），1 年 OS 为 53.1%（95% CI 45.2%-60.3%）。中位 PFS 为 5.6 个月（95% CI 4.6-7.2），中位 EFS 为 4.7 个月（95% CI 3.2-6.0），因 AE 而停止治疗的比例为 8.3%（n=15）。疗效-效果差距似乎有利于帕博利珠单抗在临床实践中的应用。
真实世界数据表明，帕博利珠单抗在先前治疗的 NSCLC 中的表现反映了临床试验结果。
