OBJECTIVES: Acute Myeloid Leukemia (AML) is a rare cancer with expensive treatments resulting in a high economic burden ($1.06 billion in the US for year 2010). Furthermore, treatment decisions in AML depend on several factors such as cytogenetics, molecular markers, and prognostic factors like patient’s age and presence of existing co-morbidities. Given the heterogeneous group of patients with different treatment pathways and outcomes, it would be of interest to see how cost-effectiveness models are structured for AML patients. The objective of this study was to review the economic evaluation studies that have assessed various treatment options given to different subgroups of AML population.

METHODS: A targeted literature search was conducted using PubMed with keywords including “acute myeloid leukemia”, “cost-effectiveness studies” and their combinations. Economic evaluation studies in English language for AML population published after 2010 were included.

RESULTS: 8 studies were included, wherein 2 studies evaluated diagnostic tests, while the others focused on different drugs used to treat AML. Markov models were used in majority of the studies (n=4) followed by partition survival models (n=3) and a combination of decision tree and Markov model (n=1). Commonly used health states included active disease, remission, and relapse. Only 2 studies included stem cell transplant (SCT) as a health state while none included risk of developing graft-versus-host disease (GVHD) which affects 80% of SCT recipients.

CONCLUSIONS: Most studies were based on data from specific clinical trials and could not address the complexity of the disease by capturing all possible outcomes. Further research is required to build a comprehensive model structure which can then be applied to general AML population. This would be helpful for the policy makers to make well-informed decisions in the future when multiple treatments for AML will need to be evaluated.