Retrospective experience of children with relapsed brain tumors treated with oral combination of axitinib and metronomic etoposide

Abstract

AbstractMetronomic chemotherapy‐based combinations have received interest for relapsed/refractory malignancies. Preclinical and clinical studies showed activity of metronomic etoposide and axitinib. We report our retrospective experience in six children treated with axitinib and metronomic etoposide for refractory/relapsed brain tumors as an “off‐label” combination. Three patients with medulloblastoma experienced partial response; one patient with atypical teratoid rhabdoid tumor (ATRT) displays an ongoing stable disease (12 months); two patients with medulloblastoma had progressive disease. Grade 3–4 toxicities were observed in two patients (thrombocytopenia, anemia, diarrhea, fatigue). The axitinib–etoposide combination shows signals of efficacy in heavily pretreated patients with relapsed/refractory brain tumors. These results were based on real‐world observation and will need formal evaluation in a phase I/II trial.