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Sequencing nearly a thousand childhood acute myeloid leukaemias showed the disease differs sharply from adult disease, with fewer mutations, more structural rearrangements and age-specific drivers, so adult genetic risk groups cannot simply be transferred to children.
Comprehensive genomic study by the TARGET initiative of 993 children and young adults with AML, including whole-genome, exome, transcriptome and methylation profiling, identifying age-specific patterns of somatic mutations, structural variants such as KMT2A and NUP98 rearrangements, and novel focal deletions.
Paediatric AML risk classification and targeted therapy development now rest on this landscape rather than on adult data.