The outcome for children with acute myeloid leukaemia (AML) continues to improve although not at the pace of acute lymphoblastic leukaemia (ALL). The current 3-year EFS and OS are 65–70% and 75–80%, respectively. The improved survival is attributed to better supportive care, refinement in risk stratification (particularly directing patients to allogeneic HCT in CR1), and improved salvage in relapsed/refractory AML (R/R AML). The enhanced understanding of the genomic landscape of paediatric AML has lead to the strategic use of novel treatments. The future of AML treatment lies in optimising the therapeutic potential of new treatments, including CAR T-cells, in combination with conventional chemotherapy and allogeneic HCT.
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