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sorafenib + azacitidine is a combination therapy being evaluated for the treatment of FLT3-ITD mutated acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). This regimen, notably studied in Phase II trials at the M.D. Anderson Cancer Center, combines the hypomethylating agent azacitidine with the multi-kinase inhibitor sorafenib. Azacitidine works as a DNA methyltransferase inhibitor, which can induce the expression of silenced genes and potentially sensitize cells to other therapies. Sorafenib targets several kinases, most importantly the FLT3-ITD mutation, which is associated with poor prognosis in AML. The combination has shown promising clinical activity, particularly in older patients or those ineligible for intensive chemotherapy.
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