Purpose of review: To describe the current management of de-novo pediatric acute myeloid leukemia (AML), excluding promyelocytic leukemia and myeloid neoplasms of patients with constitutional trisomy 21. The biology of pediatric AML, which differs from that of its adult counterpart, is briefly discussed.
Recent findings: Although survival of childhood AML has improved substantially over the past 40 years, progress has reached a plateau. Pediatric AML comprises several subtypes with diverse prognosis. Currently, about 35% of patients die of the disease, and survivors have many debilitating late effects. Clinical trials reported over the past 5 years have revealed several therapeutic concepts. First, initial intensive myelosuppressive chemotherapy is necessary to sufficiently reduce minimal residual disease and is associated with improved disease-free survival. Second, postremission chemotherapy with or without hematopoietic stem cell transplantation is necessary to eradicate AML. Third, central nervous system leukemia can be adequately managed with intrathecal chemotherapy and rarely requires radiotherapy. Finally, small differences in survival among clinical trials are explained by patient selection and quality of supportive care.
Summary: The most crucial steps for progress are greater understanding of the biology of pediatric AML and introduction of new agents targeting specific AML subtypes and age-specific factors.