Hypomethylating agents with venetoclax: have we discovered the holy grail?

Since its approval in November 2018, venetoclax with a hypomethylating agent backbone has shown promising efficacy for older, newly diagnosed acute myeloid leukemia (AML) patients who are unfit for standard intensive induction chemotherapy. This regimen is well tolerated, allows for deep and durable responses and may be increasing the prevalence of the disease. Although there is justifiable excitement, it remains to be seen to what extent venetoclax-based regimens, as they are currently administered, will have a long-term impact on the treatment of AML. This review aims to evaluate the strengths of the regimen that deserve enthusiasm as well as its shortcomings, which should be viewed as opportunities for improvement.

The clinical efficacy as well as the novel mechanism of venetoclax with hypomethylating agents will be described here.

Venetoclax with hypomethylating agents do not represent the holy grail for AML, but this regimen is a promising step in the right direction, and proof of principle that a low-intensity therapy can have a major impact on this disease.