Childhood Acute Myeloid Leukemia

Despite the use of intensive chemotherapy and hematopoietic stem cell transplantation, approximately one-third of children with acute myeloid leukemia (AML) still suffer relapse of their disease. It is unlikely that improvements in outcome can be achieved by further intensification of conventional chemotherapy. Instead, advances in the treatment of children with AML will require a greater understanding of the biology of the disease, with particular attention to the genetic abnormalities underlying leukemogenesis and drug resistance. Future clinical trials should include refined risk-directed therapy based on the genetics of the leukemic blasts and the patient’s response to therapy. More important, we must develop alternative treatment approaches, such as agents that target specific leukemia-associated abnormalities and agents that selectively eradicate leukemic stem cells.