RT Journal Article SR Electronic T1 Brain metastases from lung cancer responding to erlotinib: the importance of EGFR mutation JF European Respiratory Journal JO Eur Respir J FD European Respiratory Society SP erj01956-2009 DO 10.1183/09031936.00195609 A1 R. Porta A1 B. Massutí A1 L. Paz-Ares A1 N. Reguart A1 J.M. Sánchez A1 C. Mayo A1 P. Lianes A1 C. Queralt A1 V. Guillem A1 P. Salinas A1 S. Catot A1 D. Isla A1 A. Pradas A1 A. Gúrpide A1 J. de Castro A1 E. Polo A1 T. Puig A1 M. Tarón A1 R. Colomer A1 R. Rosell YR 2010 UL http://erj.ersjournals.com/content/early/2010/07/01/09031936.00195609.abstract AB Median survival of patients with brain metastases from non-small-cell lung (NSCLC) cancer is poor, and more effective treatments are urgently needed. We have evaluated the efficacy of erlotinib in this setting and its association with activating mutations in the EGFR gene.We retrospectively identified patients with NSCLC and brain metastases treated with erlotinib. EGFR mutations in exons 19 and 21 were analysed by direct sequencing. Efficacy and tolerability were compared according to EGFR mutational status.Sixty-nine NSCLC patients with brain metastases were identified, 17 of whom harboured EGFR mutations. Objective response rate in patients with EGFR mutations was 82.4%; no responses were observed in unselected patients (p<0.001). Time to progression within the brain for patients harbouring EGFR mutations was 11.7 months (95%CI, 7.9–15.5), compared to 5.8 months (95%CI, 5.2–6.4) for control patients, whose EGFR mutational status had not been assessed (p<0.05). Overall survival was 12.9 (95%CI, 6.2–19.7) and 3.1 months (95%CI, 2.5–3.9; p<0.001), respectively. The toxicity of erlotinib was as expected and no differences between both cohorts observed.Erlotinib is active in brain metastases from NSCLC; this clinical benefit is related to the presence of activating mutations in exons 19 or 21 of the EGFR gene.