Should acute myeloid leukemia patients with actionable targets be offered investigational treatment after failing one cycle of standard induction therapy?

Purpose of reviewTherapeutic failure in acute myeloid leukemia remains common. It may be advantageous to identify patients with suboptimal treatment responses early as they may benefit from timely care strategy changes. Here, responses portending failure of standard induction therapy are reviewed and therapeutic options examined, including use of investigational, targeted agents for suitable patients.Recent findingsPatients entering complete remission without minimal residual disease early, that is, with one cycle of standard induction chemotherapy, have a lower relapse risk and live longer than other similarly-treated patients, supporting the proposition of early complete remission without minimal residual disease as a criterion for induction therapy success. Investigational small molecule drugs are appealing for patients who fail standard therapies, but complete remission rates as a single agent are typically modest.SummaryThe relative value of different treatment strategies if a first standard induction therapy cycle fails to produce complete remission is unknown. However, retreatment with the same therapy often leads to complete remission and provides a benchmark against which other approaches should be compared. Addition of investigational small molecule drugs to standard reinduction therapy in patients with actionable targets could offer an attractive therapeutic strategy in this situation that might improve outcomes and facilitate clinical drug testing.