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Axitinib + etoposide is an investigational combination therapy consisting of two small molecule drugs with distinct mechanisms of action. Axitinib is a potent and selective inhibitor of vascular endothelial growth factor receptors (VEGFR-1, VEGFR-2, VEGFR-3), thereby blocking angiogenesis and tumor growth. Etoposide is a podophyllotoxin derivative that inhibits DNA topoisomerase II, leading to errors in DNA synthesis and apoptosis in cancer cells. This combination has been explored as an off-label regimen for relapsed or refractory brain tumors in pediatric patients, showing signals of efficacy particularly in medulloblastoma and atypical teratoid rhabdoid tumor (ATRT). The regimen typically uses metronomic dosing for etoposide alongside standard axitinib administration[1][2][3].
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