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Published on: October 10, 2025
Treatment Patterns by EGFR Mutation Status in Non-Small Cell Lung Cancer Patients in the USA: A Retrospective
Kathleen M Aguilar1, Katherine B Winfree2, Catherine E Muehlenbein3
1McKesson Specialty Health, Woodlands, TX, USA.
Introduction:
Targeted therapies, including tyrosine kinase inhibitors (TKIs) that target the sensitizing epidermal growth factor receptor (EGFR) gene are recommended for patients with non-small cell lung cancer (NSCLC). Most patients with NSCLC who test positive for the EGFR mutation and receive TKIs develop resistance to these drugs. Questions remain regarding which treatment sequence is optimal for patients with EGFR-mutant NSCLC, and few studies have evaluated patterns of TKI treatment use in NSCLC, irrespective of EGFR mutation status, in a real-world setting. This population-based study aimed to evaluate treatment patterns at a national level in the USA.
Methods:
This retrospective observational study used data from the US Oncology Network's iKnowMed database. Patients with advanced NSCLC who initiated first-line therapy with erlotinib and/or intravenous chemotherapy between January 1, 2012 and June 30, 2015 and met all other study criteria were included. Descriptive analyses assessed demographic and clinical characteristics and treatment patterns among the overall study cohort, as well as for specific erlotinib treatment subgroups, stratified by EGFR status.
Results:
Among the 3108 patients identified, 18.5% were EGFR positive, 49.8% were EGFR negative, and 31.7% were EGFR documented unknown. For the overall cohort, 18.4% received first-line erlotinib monotherapy, fewer than 1% received first-line combination therapy (erlotinib plus chemotherapy), 4.7% received second-line erlotinib monotherapy, and 3.3% received second-line combination therapy. First-line erlotinib monotherapy was used in 77.8% of all EGFR positive patients. Almost two-thirds of the overall cohort were not observed to have advanced to second-line therapy.
Conclusions:
As treatment options evolve, this study provides real-world treatment patterns that suggest concordance with NCCN guidelines and confirm the remaining need to understand sequencing of therapies and related outcomes.
Funding:
Eli Lilly and Company.
Insights
This study analyzed real-world treatment patterns for non-small cell lung cancer (NSCLC) in the US. Most EGFR-positive patients received first-line erlotinib, but many did not advance to second-line therapy.
Area of Science:
- Oncology
- Pharmacology
- Clinical Research
Background:
- Targeted therapies like tyrosine kinase inhibitors (TKIs) are recommended for EGFR-mutant non-small cell lung cancer (NSCLC).
- Resistance to TKIs is common in EGFR-mutant NSCLC patients.
- Optimal treatment sequencing for EGFR-mutant NSCLC remains unclear, with limited real-world data on TKI use patterns.
Purpose of the Study:
- To evaluate national-level, real-world treatment patterns for non-small cell lung cancer (NSCLC) in the USA.
- To assess TKI treatment use irrespective of EGFR mutation status.
- To provide insights into treatment sequencing and utilization in a large patient cohort.
Main Methods:
- Retrospective observational study using the US Oncology Network's iKnowMed database.
- Included patients with advanced NSCLC initiating first-line erlotinib and/or chemotherapy (Jan 2012–Jun 2015).
- Descriptive analyses of demographic, clinical characteristics, and treatment patterns, stratified by EGFR status.
Main Results:
- 3108 patients identified; 18.5% EGFR positive, 49.8% EGFR negative, 31.7% unknown EGFR status.
- First-line erlotinib monotherapy used in 18.4% of the overall cohort and 77.8% of EGFR-positive patients.
- Less than 1% received first-line combination therapy; ~4-5% received second-line erlotinib; nearly two-thirds did not advance to second-line therapy.
Conclusions:
- Real-world treatment patterns observed are generally consistent with NCCN guidelines.
- Further research is needed to understand optimal therapy sequencing and its impact on outcomes in NSCLC.
- The study highlights significant utilization of first-line erlotinib in EGFR-positive NSCLC but a gap in subsequent treatment escalation.
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