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Vyxeos + gemtuzumab ozogamicin is an investigational combination therapy being evaluated for the treatment of relapsed or refractory pediatric acute myeloid leukemia (AML). The regimen combines Vyxeos (CPX-351), a liposomal formulation of cytarabine and daunorubicin in a fixed 5:1 molar ratio, with gemtuzumab ozogamicin (Mylotarg), a CD33-directed antibody-drug conjugate (ADC). Vyxeos is designed to deliver synergistic ratios of chemotherapy directly to the bone marrow, where cytarabine inhibits DNA synthesis and daunorubicin inhibits topoisomerase II. Gemtuzumab ozogamicin targets CD33-positive myeloid blasts, delivering the potent cytotoxic payload calicheamicin to induce DNA double-strand breaks. This combination approach, spearheaded in clinical trials by the M.D. Anderson Cancer Center, aims to improve outcomes in difficult-to-treat pediatric AML populations by combining targeted immunotherapy with optimized liposomal chemotherapy.
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