Background: Patients with higher-risk myelodysplastic syndromes (HR-MDS), if left untreated have lower overall survival and increased economic burden. This systematic literature review (SLR) identified comprehensive evidence on the economic burden of disease among patients with low-to-high risk MDS.

Methodology: Literature search of English publications from 2011-2021 was conducted in Embase^®, MEDLINE^® and MEDLINE-In-process, including relevant conference proceedings to identify economic burden of MDS that fulfilled pre-defined inclusion criteria. Data on study characteristics, patient demographics and economic burden were extracted.

Results: 20 studies fulfilled the eligibility criteria for economic burden of MDS. Overall, costs were higher among high-risk versus low-risk MDS patients. Costs were higher in first year of diagnosis [mean (SD): $17,337 ($19,696)] versus second year [mean (SD): $12,976 ($14,135)]; chemotherapy and supportive-care costs being key cost drivers. Additionally, hospitalization, comorbidities, transfusions and hypomethylating agents (HMAs) were main cost drivers for high-risk MDS. Transfused patients incurred significantly higher mean costs than non-transfused patients ($88,824 versus $29,519, p<0.001). Transfusion independently lead to ~50% increase in monthly costs. The average (95%CI) number of transfusions over 4 years were highest for very-high-risk groups 171 (135–200) and lowest for very-low-risk groups 25 (20–32). Number of hospitalizations were higher in transfusion-dependent 6 (IQR, 4-9) than non-transfusion-dependent patients 2 (IQR, 1-4). Lack of persistence with HMAs was associated with increased total healthcare costs and resource use.

Conclusion: Overall, costs were higher with high-risk versus low-risk MDS. Transfusion and HMAs were prominent cost drivers. HMAs are the first therapeutic choice for MDS, however their usage is limited owing to lack of durable response. There exists an unmet need for appropriate therapies focusing on improving survival and reducing economic burden of MDS. These findings are suggestive, as majority of data was available from the US. More studies are needed from across geographies to draw more meaningful conclusions.