Objective: The primary objective was to examine the economic burden associated with acute myeloid leukemia (AML), a deadly hematological malignancy. AML is the most common form of acute leukemia in adults, particularly in individuals over 60 years of age; AML also accounts for 15-20% of childhood leukemia. Materials and methods: A systematic review was conducted of relevant studies published in the English language. Economic analyses of AML published between 1990 and 2002 were identified from electronic data sources using broad search criteria. Additional studies were obtained by manual searches of bibliographies of articles identified in the electronic searches. Articles were screened for relevance and included if the main theme included some element of AML cost of treatment, cost drivers, or cost-effectiveness. Studies reporting only drug prices without a formal comparison or analysis were not included. Results: Twenty-nine studies were included in the review. Although information was limited on the comprehensive economic burden of AML from a societal perspective, the costs appear to be split equally between direct and indirect costs. Direct costs of AML from a public payer perspective were available for a few countries such as the Netherlands, Sweden, US (Medicare), and Italy. These studies found that the key cost drivers appear to be hospitalization length of stay related to initial chemotherapy, relapse of disease, and bone marrow transplant (BMT) and peripheral blood stem cell transplant (PBSCT). Several cost analyses have been published comparing the different treatment strategies; however, most of them were published in the early 1990s, and their analysis revolved around cost-comparison rather than comprehensive cost-effectiveness. The published studies investigated pharmacological agents (e.g., idarubicin, daunorubicin, mitoxantrone, fludarabine and combination therapies), as well as BMT, PBSCT, and the treatment of complications. Conclusion: Studies addressing the economic costs and burden of AML are relatively sparse in the international literature. Possible reasons for such a lack of information appear to include the tow incidence rate of AML (e.g., about 260,000 new cases were reported in 2002 in the world) and the fact that it primarily afflicts older adults >60 years of age, making broad, welt-designed economic analyses a challenge for most researchers. However, due to the high cost associated with the medical procedures (e.g., BMT, PBSCT) and the aging of the world population, further research is warranted. (C) 2003 Elsevier Ltd. All rights reserved.