Late-phase investigational approaches for the treatment of relapsed/refractory acute myeloid leukemia

Acute myeloid leukemia (AML) is a malignant hematologic disorder that affects more than 13,000 adults each year in the USA. Despite continued advances in the understanding of the molecular pathogenesis of the disease and patient management, the cure rate for AML remains relatively low, largely due to a high rate of relapsed or refractory disease.

The purpose of this review is to provide an understanding of the unmet needs in relapsed/refractory AML, and to assess promising investigational agents with ongoing or planned Phase III clinical trials.

Although the treatment of relapsed/refractory AML remains a challenge, numerous new chemotherapeutics are currently in development. Enrollment in clinical trials should be strongly considered for patients with relapsed/refractory disease, and emerging data may help identify new agents with significant activity in this setting.