Erlotinib for Japanese Patients with Activating EGFR Mutation-Positive Non-Small-Cell Lung Cancer: Combined Analyses from Two Phase II Studies

AIMS: We evaluated the efficacy and safety of erlotinib, and patient characteristics affecting progression-free survival (PFS), by analyzing data from two Phase II studies of first-line erlotinib in activating EGFR mutation-positive non-small-cell lung cancer. METHODS: Data were combined from patients who received first-line erlotinib monotherapy in JO22903 (single-arm study; JapicCTI-101085) and JO25567 (randomized study; JapicCTI-111390). RESULTS: Median PFS was 10.9 months in efficacy-evaluable patients (n = 177). Major adverse events were dermatologic; no new safety signals were observed. Baseline pleural/cardiac effusion notably affected PFS (yes median 8.0 months vs no median 15.3 months) as confirmed in multivariate analysis (hazard ratio: 0.38; 95% CI: 0.25-0.58). CONCLUSION: Efficacy and safety of erlotinib monotherapy were consistent with previous studies. Baseline pleural/pericardial effusion was associated with shorter PFS.