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Background: Despite advances in our understanding of the molecular pathogenesis of acute myeloid leukemia (AML), the prognosis remains poor, mainly due to high relapse rates. Therefore, novel treatment strategies are needed to improve therapeutic outcomes for AML patients.

Background: Isocitrate dehydrogenase (IDH) is an essential enzyme in the TCA cycle. Recurrent mutations in IDH1 or IDH2 are prevalent in several cancers, including glioma and acute myeloid leukemia (AML). IDH mutations reduce cellular levels of α-ketoglutarate (α-KG), resulting in accumulation of 2-hydroxyglutarate (2-HG), an α-KG antagonist. 

Background: Although virtually all patients (pts) with AML or MDS harbor at least one somatic mutation, a minority of pts possess genetic mutations considered directly targetable by a drug. Moreover, in pts with multiple gene mutations, single-gene/single-drug matching often produces conflicting drug recommendations.

Hypomethylating agents (HMAs) (azacitidine (aza), decitabine (dec)) and lenalidomide (len) are approved agents and used to treat patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).

Background: Hypomethylating agents (HMAs) (e.g., azacitidine (aza), decitabine (dec)) and lenalidomide (len) are approved agents and used in the treatment of patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).

Background: Data from a small clinical trial of venetoclax in acute myeloid leukemia (AML) recently supported FDA breakthrough therapy designation for use in combination with hypomethylating agents in treatment-naïve patients who are ineligible for high-dose induction chemotherapy.

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