Department of Oncology, The First Affiliated Hospital of Anhui Medical University, Hefei, China.
Shanghai Lung Cancer Center, Shanghai Chest Hospital, Shanghai Jiaotong University, School of Medicine, Shanghai, China.
Lung Cancer. 2024 Nov;197:108001. doi: 10.1016/j.lungcan.2024.108001. Epub 2024 Oct 26.
With the increasing use of novel targeted drugs and immune checkpoint inhibitors (ICIs) for lung cancer (LC), the life expectancy of patients with LC has notably increased. In China, many drugs with the same mechanism of action have been approved by the National Medical Products Administration (NMPA) through phase III randomized controlled trials (RCTs). However, differences occur in these drugs' efficacy and adverse effects, all of which have been compared with standard treatments, and data from head-to-head studies are lacking.
The key RCTs of EGFR tyrosine kinase inhibitors (EGFR-TKIs), ALK-TKIs, and ICIs approved by NMPA in advanced LC in China were searched and divided into five groups. The American Society of Clinical Oncology Value Framework (ASCO-VF v2) and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS v1.1) were used to evaluate the net health benefits (NHB) of RCTs, including efficacy, adverse reactions, and patient-reported outcomes (PROs), etc. The consistency of the ASCO-VF and ESMO-MCBS was compared.
As of September 2024, 37 RCTs have been included in the ASCO-VF and ESMO-MCBS. NHB scores ranged from 12.30 to 93.25. Nineteen trials met the ASCO-VF "substantial benefit", and 28 trials achieved the ESMO-MCBS "substantial benefit". Except for icotinib, dacomitinib, and befotertinib, all EGFR-TKIs and ALK-TKIs met the threshold of two frameworks. In the ICI regimens, eight regimens met the threshold of " substantial benefit " as defined by the two frameworks and nine studies showed conflicting results. The correlation coefficient of the 37 pairs of scores in the advanced LC study was estimated to be 0.473(Spearman), and the consistency analysis showed fair agreement.(κ = 0.265, p = 0.001).
ASCO-VF and ESMO-MCBS focus on clinical efficacy and consider the adverse effects of drugs and PROs. We look forward to head-to-head studies on the different treatment options and advocate refining the ESMO-MCBS.
随着新型靶向药物和免疫检查点抑制剂（ICI）在肺癌（LC）中的应用日益增多，LC 患者的预期寿命显著延长。在中国，国家药品监督管理局（NMPA）已通过 III 期随机对照试验（RCT）批准了许多具有相同作用机制的药物。然而，这些药物的疗效和不良反应存在差异，且均与标准治疗进行了比较，缺乏头对头研究的数据。
检索并分为五组中国晚期 LC 中 NMPA 批准的 EGFR 酪氨酸激酶抑制剂（EGFR-TKI）、ALK-TKI 和 ICI 的关键 RCT。采用美国临床肿瘤学会价值框架（ASCO-VF v2）和欧洲肿瘤内科学会临床获益量表（ESMO-MCBS v1.1）评估 RCT 的净健康效益（NHB），包括疗效、不良反应和患者报告结局（PRO）等。比较 ASCO-VF 和 ESMO-MCBS 的一致性。
截至 2024 年 9 月，ASCO-VF 和 ESMO-MCBS 共纳入 37 项 RCT。NHB 评分范围为 12.30 至 93.25。19 项试验符合 ASCO-VF 的“实质性获益”标准，28 项试验符合 ESMO-MCBS 的“实质性获益”标准。除伊可替尼、达可替尼和贝福替尼外，所有 EGFR-TKI 和 ALK-TKI 均达到两个框架的阈值。ICI 方案中，8 个方案在两个框架下定义的“实质性获益”标准达到阈值，9 个研究结果存在冲突。晚期 LC 研究中 37 对评分的相关系数估计为 0.473（Spearman），一致性分析显示一致性一般（κ=0.265，p=0.001）。
ASCO-VF 和 ESMO-MCBS 侧重于临床疗效，并考虑药物的不良反应和 PRO。我们期待不同治疗方案的头对头研究，并倡导细化 ESMO-MCBS。
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