Treatment of Relapsed and Refractory AML: Intensive Approach in Fit Patients

Despite recent advances in the treatment of acute myeloid leukemia, therapy resistance manifested as relapsed or refractory disease is still common and occurs in 40–50% of younger and the great majority of elderly patients. The prognosis in relapsed/refractory (r/r)-AML is still poor with duration of relapse-free interval, age at the time point of therapy resistance, previous transplant, karyotype, and molecular markers (e.g., double mutant CEBPA, FLT3-ITD, IDH1) as important prognostic and predictive markers. Allogeneic hematopoietic cell transplantation in best case performed at the time of second complete remission remains a reliable option with curative potential, whereby the timing of allogeneic hematopoietic cell transplantation should be based on the probability to achieve a response to intensive or investigational salvage therapy. Several new approaches are currently evaluated and matching for controls may help to increase external validity of new approaches evaluated in single arm phase-II clinical trials.