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This combination therapy is an investigational regimen being evaluated by the City of Hope Medical Center for the treatment of relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The regimen consists of pembrolizumab, a humanized monoclonal antibody that functions as an immune checkpoint inhibitor by blocking the programmed death receptor-1 (PD-1) on T cells; decitabine, a hypomethylating agent that inhibits DNA methyltransferase to potentially reactivate tumor suppressor genes; and venetoclax, a small molecule inhibitor of the anti-apoptotic protein B-cell lymphoma-2 (BCL-2). By combining these agents, the therapy aims to synergistically enhance the immune system's ability to target malignant cells, reverse epigenetic silencing of genes, and directly induce apoptosis in leukemia and MDS cells. The treatment is typically administered in 42-day cycles, with pembrolizumab and decitabine given intravenously and venetoclax administered orally.
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