Advances and Future Goals in Acute Myeloid Leukaemia Therapy

The treatment of acute myeloid leukaemia (AML) remains challenging, given the disease's heterogeneous genomics, epigenetics and immunology. Although novel drugs are rapidly being developed, the outcomes of patients with AML remain suboptimal, especially among individuals older than 75 years and those with primary relapsed/refractory disease. While molecular characterization can inform the use of targeted therapies, several limitations, including low response rates and short durations of remission when targeted agents are used as monotherapies, restrict the efficacy of this strategy. It is likely that combining targeted agents with either chemotherapy or hypomethylating agents will help to advance the field. Here, we review current cytogenetic and genomic European LeukemiaNet risk-stratification models for AML. We present the cases of three patients with AML that illustrate the therapeutic recommendations for patients in specific genomic subgroups, emphasizing recent results with a hypomethylating agent plus B-cell lymphoma 2 inhibitor therapy in elderly patients. Finally, we summarize data from clinical trials that promise to improve AML therapy.