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Azacitidine + eltrombopag is a combination therapy investigated primarily for the treatment of myelodysplastic syndromes (MDS) with thrombocytopenia. Azacitidine is a hypomethylating agent that incorporates into DNA and RNA, leading to cytotoxicity in abnormal hematopoietic cells and reactivation of silenced genes through DNA hypomethylation. Eltrombopag is an oral, nonpeptide thrombopoietin receptor agonist that stimulates platelet production by activating the thrombopoietin receptor (also known as MPL). The combination was studied to address azacitidine-induced or disease-related low platelet counts in MDS patients. However, phase 3 clinical trials showed that adding eltrombopag to azacitidine did not improve hematologic outcomes and was associated with worse overall survival compared to azacitidine alone[1][6][8].
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