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Cytarabine + homoharringtonine is a combination chemotherapy regimen primarily used in the treatment of acute myeloid leukemia (AML), especially in relapsed or refractory cases. Cytarabine is a nucleoside analog that inhibits DNA synthesis, leading to cell death, particularly in rapidly dividing cells. Homoharringtonine (HHT), also known as omacetaxine mepesuccinate, is a plant alkaloid and protein translation inhibitor that blocks the elongation step of protein synthesis by binding to the 60S ribosomal subunit. This results in decreased levels of short-lived anti-apoptotic proteins such as Mcl-1 and downregulation of MYC expression, promoting apoptosis in leukemic cells[1][6][7][8]. The combination has demonstrated efficacy with manageable toxicity profiles—mainly hematologic toxicities such as pancytopenia and neutropenia—in patients with AML who have failed prior therapies[1][2]. It is also being explored for other hematologic malignancies.
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