Venetoclax-containing regimens in acute myeloid leukemia

Venetoclax in combination with hypomethylating agents (HMAs) or low-dose cytarabine (LDAC) has demonstrated exceptional activity in elderly and unfit patients with newly diagnosed acute myeloid leukemia (AML). Notably, the safety profile of venetoclax-based induction regimens was favorable, with a low rate of early treatment-related mortality, even in frail study participants. Thus, the introduction of venetoclax has transformed the landscape of AML therapy in elderly patients. Given these promising results, venetoclax in combination with other agents is now being studied as a frontline therapy in younger patients with AML, as well as in relapsed/refractory AML patients. Here, we review clinical data for venetoclax-based therapy in AML, both from prospective as well as retrospective studies, and highlight ongoing novel studies of venetoclax-containing regimens and discuss future research directions.

Automated summary

The combination of Venetoclax with hypomethylating agents or low-dose cytarabine has shown exceptional activity in elderly and unfit patients with newly diagnosed AML. The safety profile of Venetoclax-based regimens is favorable, even in frail participants, transforming the landscape of AML therapy in elderly patients. Ongoing studies are investigating the use of Venetoclax in frontline therapy for younger patients as well as in relapsed/refractory AML patients.