Objective To retrospectively evaluate the efficacy in non-small cell lung cancer (NSCLC) patients with brain metastases treated with erlotinib ± whole brain radiotherapy, and observe the adverse events. Methods From March 2012 to January 2015, 56 patients with NSCLC with brain metastases received oral erlotinib, at a dose of 150mg per day till disease progression or intolerable adverse events were developed, including 42 cases who had received brain radiotherapy.The objective response rate (ORR) and disease control rate (DCR) were observed between groups, the progression-free survival (PFS) and overall survival (OS) were calculated by Kaplan-Meier method with the use of log-rank method.Prognostic factors of the patients were analyzed with univariate and multivariate analysis in Cox regression model.Curative effect was evaluated with response evaluation criteria in solid tumors (RECIST) criteria, and adverse events with National Cancer Institute (NCI) criteria. Results ORR and DCR were 57.1%(32/56), and 82.1%(46/56), respectively. The median PFS was 12.3 months, and the OS was 8.0 months. The 1-, and 2-year PFS rates were 51.0%, and 20.0%, respectively. The 1-, and 2-year year OS rates were 36.0%, and 7.0%, respectively. Men had a higher DRR rate than women (P=0.009). Single brain metastasis patients had higher DCR rate than in multiple brain metastases patients (P=0.044). The non-smokers, less than Karnofsky (KPS) score of 70 and high recursive partitioning analysis (RPA) score groups had higher ORR (P=0.043, P 0.05). Univariate analysis showed that there were higher PFS rate and OS rate in high RPA score group than in low RPA score group (P<0.001, P<0.001), and in KPS score of 70 and more group than in less than score of 70 group (P<0.001, P<0.001), respectively. The non-smokers and patients with adenocarcinoma group had longer OS times. Multivariate analysis showed that KPS score was the independent prognostic factor of PFS and OS (P<0.001, P=0.005), pathological type was the independent prognostic factor of PFS (P=0.001). Conclusions Erlotinib is effective and safe for NSCLC patients with brain metastases. Men and the single brain metastasis patients, non-smokers, high KPS and RPA score groups had higher DCR rates. KPS score is the most important influencing factors for patient's PFS and OS time. Women and the patients with adenocarcinoma have higher PFS rate than those of men and squamous cell carcinoma. Key words: Quinazolines/AD; Radiotherapy; Carcinoma, non-small-cell lung/CO/TH; Brain neoplasms/SC/TH
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