Acute myeloid leukaemia (AML) is characterized by a block in differentiatio
n and an unregulated proliferation of myeloid progenitor cells. While the c
ause of AML in children is unknown, risk factors that have been identified
include exposure to toxins such as ethanol, pesticides and dietary topoisom
erase II inhibitors, prior chemotherapy with alkylating agents or topoisome
rase II inhibitors, constitutional disorders such as Down's syndrome and ty
pe I neurofibromatosis, and haematopoietic failure syndromes such as Fancon
i anaemia and severe congenital neutropenia. With intensified chemotherapy
including high-dose Ara-C, followed in many cases by bone marrow transplant
ation, and with improvements in supportive care, current survival rates app
roach 50%. Future advances in paediatric AML will include better risk strat
ification to determine optimal treatment and targeted cytotoxic therapy.