Childhood acute myeloid leukaemia

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  • Jeffrey E. Rubnitz
    Department of Oncology St. Jude Children's Research Hospital Memphis TN USA
  • Hiroto Inaba
    Department of Oncology St. Jude Children's Research Hospital Memphis TN USA

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<jats:title>Summary</jats:title><jats:p>Although acute myeloid leukaemia (<jats:styled-content style="fixed-case">AML</jats:styled-content>) has long been recognized for its morphological and cytogenetic heterogeneity, recent high‐resolution genomic profiling has demonstrated a complexity even greater than previously imagined. This complexity can be seen in the number and diversity of genetic alterations, epigenetic modifications, and characteristics of the leukaemic stem cells. The broad range of abnormalities across different <jats:styled-content style="fixed-case">AML</jats:styled-content> subtypes suggests that improvements in clinical outcome will require the development of targeted therapies for each subtype of disease and the design of novel clinical trials to test these strategies. It is highly unlikely that further gains in long‐term survival rates will be possible by mere intensification of conventional chemotherapy. In this review, we summarize recent studies that provide new insight into the genetics and biology of <jats:styled-content style="fixed-case">AML</jats:styled-content>, discuss risk stratification and therapy for this disease, and profile some of the therapeutic agents currently under investigation.</jats:p>

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