Therapy for childhood acute myeloid leukemia: role of allogeneic bone marrow transplantation.

Acute myeloid leukemia (AML) has one of the lowest survival rates of childhood cancers. The first significant improvement in AML therapy started with the introduction of the now standard regimen of 3 days of anthracyclines and 7 days of cytarabine (Ara-C), the so-called 3+7 combinations. Several different therapeutic approaches have been taken in attempts to improve the outcome, including intensification of therapy both for remission induction and in the postremission phase. Intensification of postremission therapy included multiple courses of high-dose chemotherapy and/or myeloablative therapy followed by stem- cell rescue from either allogeneic or autologous sources. Furthermore, risk-tailored therapy is now possible, by cytogenetic risk stratification, promptness of remission induction, and identification of distinct clinical subgroups such as children with Down syndrome. This approach is rapidly changing potential therapeutic strategies for children with AML. It is in this changing mileu that we address the proper role of stem-cell transplantation, a modality that is changing (like chemotherapy) with expanding stem-cell sources and approaches to decrease transplant-related toxicity.