New therapeutic strategies for high-risk acute myeloid leukemia

Treatments for acute myeloid leukemia (AML) had remained essentially unchanged for several years; however, the advent of molecular testing has generated insight into the biology of this disease which is now being translated into clinical practice. New treatment strategies which improve drug delivery and exploit cellular targets are changing the landscape of how we treat this disease.

Induction therapy is in the process of changing for several patient populations. The introduction of CPX-351 offers a novel strategy for treating patients with therapy-related AML or AML with myelodysplasia-related changes; gemtuzumab ozogamicin may become incorporated into standard induction therapy, especially for patients with core-binding factor leukemias; and for older adults, combination therapy with venetoclax may offer a more efficacious strategy than the single-agent regimens previously used. Additionally, targeted therapies are now becoming available for patients with mutations in FMS-like tyrosine kinase 3 (FLT3) or isocitrate dehydrogenase 2 (IDH2), ushering in an era of personalized medicine in the treatment of AML.

The US Food and Drug Administration approval of several agents in 2017 will change the way AML treatment is approached and will offer both clinicians and patients a new armamentarium with which to treat this disease.