State Key Laboratory of Molecular Oncology, Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Thorac Cancer. 2022 May;13(9):1289-1298. doi: 10.1111/1759-7714.14380. Epub 2022 Mar 28.
There is a lack of clinically available predictive models for patients with epidermal growth factor receptor (EGFR) mutation positive, advanced non-small cell lung cancer (NSCLC) treated with EGFR-tyrosine kinase inhibitors (TKIs).
The clinical data of patients at the Cancer Hospital, Chinese Academy of Medical Sciences between from January 2016 to January 2021 were retrospectively retrieved as training set. The patients from BENEFIT trial were for the validation cohort. The nomogram was built based on independent predictors identified by univariate and multivariate Cox regression analyses. The discrimination and calibration of the nomogram were evaluated by C-index and calibration plots.
A total of 502 patients with complete clinical data and follow-up information were enrolled in this study. Five independent prognostic factors, including The Eastern Cooperative Oncology Group Performance Status scale (ECOG PS), EGFR mutation subtype, EGFR co-mutation, liver metastasis and malignant pleural effusion (p < 0.05). The C-indexes of the nomogram were 0.694 (95% confidence interval [CI], 0.663-0.725) for the training set and 0.653 (95% CI, 0.610-0.696) for the validation set. The calibration curves for the probabilities of 9-, 12- and 18-month progression-free survival (PFS) revealed satisfactory consistency in both the internal and external validations. Additionally, the patients were divided into two groups according to risk (high-risk, low-risk), and significant differences in PFS were observed between the groups in the training and external validation cohorts (p < 0.001).
We constructed and validated a convenient nomogram that have the potential to become an accurate and reliable tool for patients with EGFR mutation positive, advanced NSCLC to individually predict their potential benefits from EGFR-TKIs, and facilitate clinical decision-making.
目前缺乏针对表皮生长因子受体(EGFR)突变阳性的晚期非小细胞肺癌(NSCLC)患者接受 EGFR 酪氨酸激酶抑制剂(TKI)治疗的临床可用预测模型。
回顾性收集中国医学科学院肿瘤医院 2016 年 1 月至 2021 年 1 月期间的患者临床资料作为训练集。BENEFIT 试验患者为验证队列。基于单因素和多因素 Cox 回归分析确定的独立预测因素构建列线图。通过 C 指数和校准图评估列线图的区分度和校准度。
共纳入 502 例具有完整临床数据和随访信息的患者。5 个独立预后因素包括东部肿瘤协作组体力状态评分(ECOG PS)、EGFR 突变亚型、EGFR 共突变、肝转移和恶性胸腔积液(p<0.05)。列线图在训练集和验证集的 C 指数分别为 0.694(95%可信区间[CI],0.663-0.725)和 0.653(95%CI,0.610-0.696)。内部和外部验证的 9、12 和 18 个月无进展生存期(PFS)概率校准曲线显示出良好的一致性。此外,根据风险(高风险、低风险)将患者分为两组,在训练集和外部验证队列中,两组的 PFS 差异有统计学意义(p<0.001)。
我们构建并验证了一个方便的列线图,它有可能成为一种准确可靠的工具,用于预测 EGFR 突变阳性的晚期 NSCLC 患者接受 EGFR-TKI 治疗的潜在获益,并为临床决策提供帮助。