A relevant number of patients with acute myeloid leukemia (AML) do not receive active treatment after diagnosis. This analysis aims to understand how much the cost directly related to treatment represents in the total cost of patient's management. As secondary objectives, to assess the economic impact of achieving remission vs. non-remission, and the burden of non-treated disease.

A systematic review was conducted in June 2023 through Cochrane Collaboration, Medline, EMBASE, CRD, and Lilacs databases. Inclusion criteria considered experimental or observational studies with data on healthcare resource utilization (HRU) and/or costs of AML management, describing at least one of the predefined outcomes. Modeling-based studies were excluded. Primary outcomes were % of disease-specific treatment costs (DSTC) in total direct medical costs and % of antineoplasic costs (ANC) in total direct medical costs. As secondary outcomes, costs per patient per month (PPPM) of achieving remission vs. non-remission, and HRU and costs among those not receiving active treatment (total cost, % of transfusions and length of stay in hospital). Only direct medical costs were considered.

Searches returned 1,681 titles, 46 complete articles were evaluated, and 11 was selected (all observational retrospective studies). Eight were conducted in United States, one in Spain, one in Netherlands, and one in 22 countries (Asian-Pacific, Europe, Canada, Middle East, Africa, and Latin America regions). Data were collected from 1991 to 2020. Sample sizes ranged from 202 to 39,568 and mean ages from 52.8 to 78.5 years in studies. Percentage of DSTC ranges from 51.4% to 73.6%, including all costs registered as directly related to AML. Percentage of ANC varied from 2.8% to 37.3% (the highest value also included hospital costs). Costs for patients in non-remission were 1.65 to 7.34 times higher than those in remission. Furthermore, remission for prolonged periods exhibited lower PPPM costs vs. shorter durations. Regarding patients without active treatment, management costs ranged from 4,854 USD to 8,596 USD PPPM. Blood product transfusions occurred in 26.3% to 71% of them and they remained 8 to 15.4 days hospitalized. One study showed infection as the main reason for hospitalization (49%), followed by transfusion (36%), and progression/relapse (23%). Another cited 72% of patients without active treatment receiving antibiotics or antivirals, 34% using antifungals, and 7% needing growth factors infusion. In all studies, hospitalization was the main cost driver among all cost components, reaching more than 70% of the total.

According to global literature, despite the availability of active treatments, many AML patients remain untreated. Thus, this review described such economic impacts. Results showed that costs directly related to treatment do not represent the largest component driver. In addition, those without active treatment incur high HRU and costs. The main limitation is the heterogeneity regarding age, disease stage, paying sources, active treatment regimen and follow-up time among studies.

The absence of active treatment and lower remission rates showed a significant financial impact on healthcare systems, highlighting the need for greater focus on those topics to improve survival and optimize costs in AML context.

AbbVie sponsored the study; contributed to design and interpretation of data; in reviewing and approval of abstract. Natália Santoni, Rafael Coutinho, Ingrid Pereira are employees of AbbVie and Rafael Coutinho also owns AbbVie stock. Roberta Arinelli, Carolina Padula are external authors who contributed to this research, having no conflicts of interest with AbbVie.