Abstract

Simple SummaryNo comprehensive and simple prognostic model based on pretreatment factors exists for patients with epidermal growth factor receptor mutation-positive (EGFRm+) non-small cell lung cancer (NSCLC) undergoing EGFR-tyrosine kinase inhibitors (EGFR-TKIs). A total of 11 independent prognostic factors were identified by multivariate analysis, including performance status, morphology, mutation, stage, EGFR-TKIs, and metastasis to liver, brain, bone, pleura, adrenal gland, and distant lymph nodes. We established a nomogram based on independent pretreatment factors and used it to stratify EGFRm+ NSCLC patients undergoing EGFR-TKI treatment into five different risk groups for survival using recursive partitioning analysis. The performance of this nomogram was good and feasible, providing clinicians and patients with additional information for evaluating therapeutic options.Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) are the standard treatment for EGFR mutation-positive (EGFRm+) non-small cell lung cancer (NSCLC). This study aimed to create a novel nomogram to help physicians suggest the optimal treatment for patients with EGFRm+ NSCLC. Records of 2190 patients with EGFRm+ NSCLC cancer who were treated with EGFR-TKIs (including gefitinib, erlotinib, and afatinib) at the branches of a hospital group between 2011 and 2018 were retrospectively reviewed. Their clinicopathological characteristics, clinical tumor response, progression-free survival (PFS), and overall survival (OS) data were collected. Univariate and multivariate analyses were performed to identify potential prognostic factors to create a nomogram for risk stratification. Univariate analysis identified 14 prognostic factors, and multivariate analysis confirmed the pretreatment independent factors, including Eastern Cooperative Oncology Group performance status, morphology, mutation, stage, EGFR-TKIs (gefitinib, erlotinib, or afatinib), and metastasis to liver, brain, bone, pleura, adrenal gland, and distant lymph nodes. Based on these factors, a novel nomogram was created and used to stratify the patients into five different risk groups for PFS and OS using recursive partitioning analysis. This risk stratification can provide additional information to clinicians and patients when determining the optimal therapeutic options for EGFRm+ NSCLC.

Highlights

  • Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) have long been the standard treatment for EGFR mutation-positive (EGFRm+) non-smallcell lung cancer (NSCLC), the responses to EGFR-TKIs and the duration of treatment are variable

  • Due to considerations regarding cost-effectiveness and the toxicities associated with these regimens, 1G/2G TKI monotherapy remains widely used in clinical practice, whereas these other regimens may serve as alternative options for EGFRm+ non-small cell lung cancer (NSCLC) patients

  • A total of 2190 EGFRm+ NSCLC patients treated with 1G/2G EGFR-TKIs as firstline treatment were analyzed in this study, including 1059 patients treated with gefitinib, 496 with erlotinib, and 635 with afatinib

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Summary

Introduction

Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) have long been the standard treatment for EGFR mutation-positive (EGFRm+) non-smallcell lung cancer (NSCLC), the responses to EGFR-TKIs and the duration of treatment are variable. The median progression-free survival (PFS) for first-generation (1G: gefitinib and erlotinib) [1,2,3,4] and second-generation (2G: afatinib, dacomitinib) [5,6,7,8] TKIs is approximately 12 months. Frontline osimertinib [11,12], gefitinib plus chemotherapy [13], erlotinib plus bevacizumab [14], and erlotinib plus ramucirumab [15] are new treatment strategies due to demonstrated improvements in PFS and overall survival (OS) based on phase III studies. Due to considerations regarding cost-effectiveness and the toxicities associated with these regimens, 1G/2G TKI monotherapy remains widely used in clinical practice, whereas these other regimens may serve as alternative options for EGFRm+ NSCLC patients. The rapid and accurate identification of patients with a high risk of progression who may benefit from more aggressive treatment represents a currently unmet need in daily oncological practice

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