azacitidine + arsenic trioxide

A combination regimen of the hypomethylating agent azacitidine and the inorganic antineoplastic arsenic trioxide investigated primarily for myelodysplastic syndromes and acute myeloid leukemia. Azacitidine inhibits DNA methyltransferases leading to DNA hypomethylation and incorporates into RNA/DNA causing cytotoxicity[4]. Arsenic trioxide induces apoptosis and differentiation in leukemic cells; in APL it is highly efficacious[8][1]. Preclinical work shows azacitidine can sensitize AML cells to arsenic trioxide by demethylating the HNF1A promoter, upregulating AQP9, increasing intracellular arsenic uptake, and enhancing cytotoxicity[1][2][3]. A clinical study explored the schedule of azacitidine (SC, days 1–5) with arsenic trioxide (IV, intermittent dosing) in MDS/CMML patients[5]. Safety considerations include arsenic trioxide–associated QTc prolongation risk, requiring ECG/electrolyte monitoring[6].

Other names

azacytidine + arsenic trioxide