Acute Myeloid Leukemia: Is That All There Is?

Acute myeloid leukemia (AML) is characterized by the clonal proliferation of malignant myeloid blast cells in the marrow along with impaired normal hematopoiesis. With an almost stagnant approach for the management of patients with AML in the last three decades, the main purpose of this paper is to increase our understanding of recent scientific advancements for the enhanced diagnosis and treatment of AML. Existing research data related to different approaches for a possible improvement in AML management has been collected and discussed. The identification of recurrently mutated genes, such as CCAAT-enhancer-binding proteins α (CEBPα), Fms-related tyrosine kinase 3 (FLT3), and nucleophosmin 1 (NPM1) along with the classic diagnostic karyotype has improved prognostic-risk stratification. Moreover, mutations affecting cellular metabolism like isocitrate dehydrogenase (IDH1), lysine-specific demethylase 1 (LSD 1), and nuclear factor kappa-light-chain-enhancer of activated B cells (NF-κB) have become a huge success by providing targets for novel therapeutic drugs. Checkpoint inhibitors (CPI) and vaccination against tumor-associated antigen are added options considered, which require further trials before their efficacy can be determined. An important tool in monitoring early response to therapy, minimal residual disease (MRD) assays can be further refined by including pretreatment parameters such as cytogenetic and molecular markers. Potential side effects and resistance to treatment remains a huge barrier in completely finding success against AML and work needs to be done to find combinations of immunotherapies to possibly reduce adaptive resistance by AML.