Intracavitary chemotherapy with epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI) is not superior to TKI monotherapy in controlling malignant pleural effusion recurrence in EGFR-mutated lung cancer patients.

Epidermal growth factor receptor (EGFR) mutated non-small cell lung cancer (NSCLC) patients benefit from EGFR-tyrosine kinase inhibitors (TKIs) therapy. There are few studies comparing the efficacy between intrapleural chemotherapy combination with TKIs and TKIs alone in controlling re-accumulation of malignant pleural effusions (MPEs). The purpose of the study was to determine if patients with EGFR-mutated NSCLC and MPEs would benefit from intrapleural chemotherapeutics with an oral EGFR-TKI than EGFR-TKI alone.

We evaluated EGFR-mutated lung cancer patients with MPEs in Zhejiang Cancer Hospital. We evaluated the efficacy. Progression-free survival (PFS) and overall survival (OS) was evaluated by Kaplan-Meier method.

One hundred one NSCLC patients with MPEs at the time of diagnosis were included. We divided the patients into two groups. The overall response rate (ORR) with respect to MPE recurrence between the TKI alone and combination therapy groups was 65.5% (38/58) and 58.1% (25/43) (P=0.449). The disease control rate was 89.7% (52/58) and 86.0% (37/43) (P=0.579), respectively. The PFS in the TKI alone and TKI plus intrapleural drugs was 10.3 and 9.9 months, respectively (P=0.746). The intrapleural PFS was 11.4 and 11.0 months for the TKI alone and combination groups, respectively (P=0.188). The OS was 24.9 and 22.6 months (P=0.543), respectively. Hematologic toxicity and chest pain were more frequent in the combination therapy than TKI alone groups.Intrapleural chemotherapy with TKI did not improve the efficacy of controlling MPEs in patients with EGFR-mutated NSCLC, but may increase adverse events, which are typical side effects of chemotherapy. We could treat these patients with TKI drugs alone combined with pleural effusion drainage.