Real-World Data Of Osimertinib In Patients With Pretreated Non-Small Cell Lung Cancer: A Retrospective Study
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Updated: Jan 2, 2026

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
Published on: November 22, 2021
Yuxin Mu1, Puyuan Xing1, Xuezhi Hao1
1National Cancer Center, National Clinical Research Center for Cancer, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, People's Republic of China.
Purpose:
Osimertinib is an oral, irreversible epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) targeted for both EGFR sensitizing mutations and T790M resistance mutation in patients with non-small-cell lung cancer (NSCLC). We assessed efficacy and safety of osimertinib in patients with pretreated NSCLC in a real-world setting.
Patients And Methods:
Ninety-four patients with advanced NSCLC who received osimertinib after progression of prior EGFR-TKIs or chemotherapy treatments were retrospectively collected.
Results:
In patients evaluable for response analysis (n = 91), overall objective response rate (ORR) was 47.3%, and disease control rate (DCR) was 90.1%. Median duration of response (DoR) in responding patients was 12.5 months (95% confidence interval [CI], 10.7 to 14.3). Median progression-free survival (PFS) was 8.5 months (95% CI, 7.4 to 9.6) in 2nd line group, 9.1 months (95% CI, 6.6 to 11.6) in ≥3rd line group, and 8.6 months (95% CI, 7.2 to 10.0) in overall population. For subgroup analysis, DCR and median PFS were 91.9% and 8.6 months (95% CI, 7.2 to 10.0) in patients with detectable T790M mutation, respectively, while 80.0% and 3.2 months (95% CI, 0.5 to 5.9) for those without. Median PFS was significantly longer for T790M-positive patients co-occurring with exon19del than with L858R (17.9 months vs 7.3 months; P<0.001). Among 45 patients with metastases to the central nervous system (CNS), median systemic PFS was 8.8 months (95% CI, 6.9 to 10.7), while intracranial time to progression (iTTP) was not reached. Safety profile was acceptable, no adverse events (AEs) related deaths was observed.
Conclusion:
Osimertinib was highly active in patients with pretreated advanced NSCLC who harbored EGFR T790M mutation, with manageable side-effects.
Insights
Osimertinib demonstrated significant efficacy in pretreated non-small-cell lung cancer (NSCLC) patients with EGFR T790M mutations. The drug showed a high response rate and manageable side effects in this real-world setting.
Area of Science:
- Oncology
- Pharmacology
Background:
- Non-small-cell lung cancer (NSCLC) remains a leading cause of cancer-related deaths worldwide.
- Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) have revolutionized NSCLC treatment.
- Acquired resistance to first- and second-generation EGFR-TKIs, often mediated by the T790M mutation, presents a significant clinical challenge.
Purpose of the Study:
- To evaluate the real-world efficacy and safety of osimertinib in patients with pretreated advanced NSCLC.
- To assess the treatment outcomes in patients with EGFR T790M resistance mutations after prior EGFR-TKI or chemotherapy treatments.
Main Methods:
- A retrospective analysis was conducted on 94 patients with advanced NSCLC who received osimertinib post-progression on prior therapies.
- Data collected included objective response rate (ORR), disease control rate (DCR), duration of response (DoR), progression-free survival (PFS), and safety profiles.
- Subgroup analyses were performed, including stratification by T790M mutation status and specific EGFR mutations (exon19del, L858R), as well as patients with central nervous system metastases.
Main Results:
- The overall objective response rate (ORR) was 47.3% and the disease control rate (DCR) was 90.1% in evaluable patients (n=91).
- Median progression-free survival (PFS) was 8.6 months in the overall population, with longer PFS observed in patients with detectable T790M mutations (8.6 months) compared to those without (3.2 months).
- Median PFS was significantly longer for T790M-positive patients with exon19del compared to those with L858R (17.9 months vs 7.3 months; P<0.001). Intracranial time to progression was not reached in patients with CNS metastases.
Conclusions:
- Osimertinib demonstrates high activity and a manageable safety profile in pretreated advanced NSCLC patients, particularly those harboring the EGFR T790M mutation.
- The real-world data support the efficacy of osimertinib in a diverse NSCLC patient population, including those with central nervous system involvement.
- Osimertinib represents a valuable therapeutic option for patients who have progressed on prior EGFR-TKI therapies.
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