Relapsed/refractory acute myeloid leukemia: any progress?

Purpose of review: Aim of this review was to focus on prognostic and predictive factors, standard and new treatment approaches, and on statistical considerations for future clinical trials in patients with relapsed/refractory acute myeloid leukemia (r/r-AML). Recent findings: New prognostic molecular markers were identified in r/r-AML, FLT3-ITD, mutated IDH1, and biallelic CEBPA mutations. Intensive combination chemotherapy including gemtuzumab ozogamicin emerged as an effective salvage therapy in refractory AML. Timing of allo-HCT in r/r-AML may be oriented at the probability to achieve a response to intensive salvage therapy. Several new treatment approaches ranging from new and modified cytotoxic drugs to targeted approaches are in clinical development with first efficacy assessment in single-arm phase II studies. Their external validity may be considerably increased by using a novel design based on a matching approach. Summary: FLT3-ITD, mutated IDH1, and biallelic CEBPA mutations are identified as prognostic molecular markers in r/r-AML. Timing of allo-HCT should be based on the probability to achieve a response to intensive salvage therapy. Several new approaches are currently evaluated and matching for controls may help to increase external validity.

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