Clinical outcomes after first-line EGFR inhibitor treatment for patients with NSCLC, EGFR mutation, and poor performance status
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Updated: May 6, 2026

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
Published on: November 22, 2021
Yusuke Okuma1, Yukio Hosomi, Makoto Nagamata
1Department of Thoracic Oncology and Respiratory Medicine, Tokyo Metropolitan Cancer and Infectious diseases Center Komagome Hospital, 3-18-22 Honkomagome, Bunkyo, Tokyo 113-8677, Japan. y-okuma@cick.jp.
Background:
The phase II NEJ001 trial suggested that gefitinib was active against advanced non-small cell lung cancer (NSCLC) even in patients with poor performance status (PS). Clinical response among the patients harboring epidermal growth factor receptor (EGFR) mutation with poor PS is fair; however, gefitinib does not have as much continued efficacy as in patients with good PS. This study has retrospectively investigated the clinical outcomes of gefitinib treated patients with advanced NSCLC, EGFR mutations, and poor PS.
Patients And Methods:
A total of 208 patients with advanced NSCLC and poor PS treated with gefitinib from 2004 to 2013 were retrospectively evaluated. Outcomes were studied after stratification for gender, smoking status, histological subtype, and EGFR mutation status.
Results:
Fifty-two patients (25.0%) with advanced NSCLC, EGFR mutation, and poor PS were treated with gefitinib. The overall response rate was 65.4%. The median progression-free survival, median survival time, and one-year survival rate was 6.6 months, 19.6 months, and 62.9%, respectively. Death due to interstitial lung disease occurred in 11.5% of the patient population. In multivariate analysis, a PS of 4 was independently associated with poor outcomes (hazard ratio=10.5; 95% Confidence interval=1.92-50.19; p=0.0091).
Conclusion:
Patients with advanced NSCLC, EGFR mutation, and poor PS have poor outcomes in response to gefitinib. However, the indication of gefitinib for such patients will not be changed in clinical practice and oncologists should treat these patients with more careful follow-up since for those with poor PS, therapy may be more toxic than for patients with good PS.
Insights
Gefitinib shows limited efficacy in advanced non-small cell lung cancer (NSCLC) patients with poor performance status (PS) and EGFR mutations. Careful monitoring is advised due to potential toxicity in this vulnerable patient group.
Area of Science:
- Oncology
- Pharmacology
Background:
- Gefitinib showed activity in advanced non-small cell lung cancer (NSCLC) patients, including those with poor performance status (PS).
- Efficacy of gefitinib may be limited in patients with EGFR mutations and poor PS compared to those with good PS.
- This study retrospectively analyzed gefitinib outcomes in advanced NSCLC patients with EGFR mutations and poor PS.
Purpose of the Study:
- To retrospectively investigate the clinical outcomes of gefitinib treatment in advanced NSCLC patients with EGFR mutations and poor performance status.
- To evaluate the efficacy and safety of gefitinib in a specific subgroup of NSCLC patients.
Main Methods:
- Retrospective evaluation of 208 advanced NSCLC patients treated with gefitinib (2004-2013).
- Outcomes analyzed after stratification by gender, smoking status, histological subtype, and EGFR mutation status.
- Multivariate analysis was performed to identify independent prognostic factors.
Main Results:
- Fifty-two patients (25.0%) with advanced NSCLC, EGFR mutation, and poor PS received gefitinib.
- Overall response rate was 65.4%, with median progression-free survival of 6.6 months and median survival of 19.6 months.
- A PS of 4 was independently associated with poor outcomes (HR=10.5; p=0.0091), and interstitial lung disease caused death in 11.5% of patients.
Conclusions:
- Patients with advanced NSCLC, EGFR mutation, and poor PS exhibit poor outcomes with gefitinib.
- Despite poor outcomes, gefitinib indication remains unchanged, but requires careful follow-up due to increased toxicity in poor PS patients.
- Oncologists should exercise caution and provide closer monitoring for this patient group.
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