Pediatric Acute Myeloid Leukemia Biology and Therapeutic Implications of Genomic Variants

Acute myeloid leukemia (AML) is a molecularly heterogeneous disease and age-associated molecular alterations result in younger children harboring a distinct signature from older children and adolescents. Pediatric AML has a genetic and epigenetic profile with significant differences compared to adult AML. Somatic and epigenetic alterations contribute to myeloid leukemogenesis and can evolve from diagnosis to relapse. Cytogenetic alterations, somatic mutations and response to induction therapy are important in informing risk stratification and appropriate therapy allocation. Next-generation sequencing technologies are providing novel insights into the biology of AML and have the ability to identify potential targets for therapeutic intervention.