Acute myeloid leukaemia

Abstract

For several decades, few substantial therapeutic advances have been made for patients with acute myeloid leukaemia. However, since 2017 unprecedented growth has been seen in the number of drugs available for the treatment of acute myeloid leukaemia, with several new drugs receiving regulatory approval. In addition to advancing our therapeutic armamentarium, an increased understanding of the biology and genomic architecture of acute myeloid leukaemia has led to refined risk assessment of this disease, with consensus risk stratification guidelines now incorporating a growing number of recurrent molecular aberrations that aid in the selection of risk-adapted management strategies. Despite this promising recent progress, the outcomes of patients with acute myeloid leukaemia remain unsatisfactory, with more than half of patients ultimately dying from their disease. Enrolment of patients into clinical trials that evaluate novel drugs and rational combination therapies is imperative to continuing this progress and further improving the outcomes of patients with acute myeloid leukaemia.

Figure 1:. Management of acute myeloid leukaemia in adults

HLA=human leukocyte antigen. HSCT=haemopoietic stem cell transplant.

Figure 2:. Examples of selected novel therapeutic strategies in acute myeloid leukaemia

Five therapeutic targets being developed for the management of acute myeloid leukaemia are highlighted, as well as representative compounds that are in clinical trials. These examples are not intended to be all inclusive. Strategies that eliminate leukaemia stem cells, which are generally chemoresistant and serve as a reservoir for relapse, are needed to achieve cure.