Erlotinib as First-Line Treatment for Patients with Advanced EGFR Mutation-Positive Non-Small-Cell Lung Cancer: Phase Iiib Study

Abstract

Aim: Erlotinib has been shown to improve progression-free survival when given as first-line treatment for patients with non-small-cell lung cancer (NSCLC) with activating EGFR mutations. We aimed to assess the efficacy and safety of erlotinib for first-line treatment of patients with advanced EGFR-mutation positive NSCLC. Methods: This is an phase IIIb study of erlotinib treatment conducted during 2012 to 2014 at 4 participating hospitals in Serbia. Eligible participants were adults (>18 years) with NSCLC and EGFR mutations (exon 19 deletion or L858R mutation in exon 21) with no history of chemotherapy for metastatic disease. The patients were referred to receive oral erlotinib 150 mg per day. The primary endpoint was progression-free survival (PFS) in the intention-to-treat population. PFS was defined as the time from the first dose to the date of first occurrence of disease progression or death. Response rate was based on response measured according to RECIST criteria. The safety was assessed in all patients who received study drug (≥1 dose). Results: 30 patients with EGFR mutations, mean age 62.2 ± 9.7 years, 60.0% women, ex/current smokers 53.3%, were enrolled in the study. 86.2% of patients had stage IV disease, with ECOG ≤1 in 96.7% of patients. Exon 19 deletion and exon 21 mutation had 62.1% and 37.9% patients, respectively. At data cutoff (Jan 5, 2014), median PFS was 8.3 months (95% CI 4.0-12.5). 25 (83.3%) patients finished the study, 22 (73.3%) due to progressive disease and 1 due to death. One patient had treatment-related severe adverse event and one patient was lost to follow-up. A response rate of 26.7% is obtained, with one patient in complete remission after 22 months. Conclusions: Routine baseline tissue-based assessment of EGFR mutations in patients with NSCLC can be used to identify the subgroup of patients suitable for safe and effective treatment with EGFR tyrosine-kinase inhibitors.