OBJECTIVES: Patients with FLT3-mutated AML have poor prognoses due to short survival, high incidence of relapse, and a lack of effective treatment options. The objective of this study was to review AML-related literature and estimate the economic burden of FLT3-mutated AML in the United States. METHODS: A systematic literature review was conducted to identify relevant publications from 2000-2011. Estimates of the epidemiology of FLT3-mutated AML were calculated from SEER data and US Census projections. An Excel model was developed to estimate the BOI of FLT3-mutated AML.  Resource utilization estimates were obtained from the literature and expert opinion. RESULTS: A total of 607 citations were identified and 35 articles abstracted.  No direct estimates for the incidence or prevalence of FLT3-mutated AML in the United States were identified in the literature. The prevalence of AML in 2008 was obtained from SEER data (27,813 patients aged ≥20 years). In 2010, it was estimated that 784 adults <60 years and 1,622 adults ≥60 years were diagnosed with FLT3-mutated AML. The BOI of FLT3-mutated AML in the US was estimated at $251 million in 2010, including $191 million in direct costs and $60 million in lost productivity. For newly diagnosed patients <60 years, the burden of FLT3-mutated AML was estimated at $90 million in 2010. Inpatient hospitalizations accounted for 36% of direct costs and stem cell transplants for 38%. The cost per newly diagnosed FLT3-mutated AML patient <60 years was estimated at $114,193 compared to $105,819 for patients with non-FLT3-mutated AML. As early mortality and early retirement were not included in indirect costs, these may be underestimated. CONCLUSIONS: FLT3-mutated AML potentially represents a greater per-patient burden than non-FLT3-mutated AML due to shorter survival and greater use of stem cell transplants. Investigational treatments targeting the FLT3 mutation may provide an additional therapeutic option and have the potential to improve clinical outcomes.