Emerging treatments in acute myeloid leukemia: current standards and unmet challenges

Acute myeloid leukemia (AML) is rare and difficult to treat. Although remission is achieved in most patients with newly diagnosed disease, relapse occurs in most cases. For more than 40 years, the standard up-front induction treatment has been a combination of continuous-infusion cytarabine and an anthracycline. Risk stratification by molecular and cytogenetic characteristics and measurable residual disease (MRD) status informs decisions regarding referral to consolidative allogeneic hematopoietic cell transplant. In 2017, for the first time in years, 4 drugs are under consideration for approval by the US Food and Drug Administration. One of these agents, the multikinase inhibitor midostaurin, has already been approved for the treatment of patients with FLT3-mutated AML. The heterogeneity of AML suggests that single-target agents are unlikely to succeed at curing large numbers of patients, and that combinations of novel agents and traditional chemotherapy will be required to achieve this goal. Additionally, the 2017 European LeukemiaNet criteria suggest that treating physicians should strive for the new, stringent remission category (ie, complete remission without MRD). How the new drug approvals in 2017 will change practice is not clear, and further work remains to be done in treating and curing patients with AML.