OBJECTIVES: Individuals with sickle cell disease (SCD), β-thalassaemia or myelodysplastic syndromes (MDS) commonly require red blood cell transfusions, increasing their risk of transfusional iron overload. In individuals with SCD, β-thalassaemia, or MDS, both the underlying condition and the treatment regimens can have a significant effect on health related quality of life (HRQoL) and costs, with indirect costs arising from presenteeism, absenteeism, and non-work related productivity losses. The aim of the present study was to review and summarize the literature on the economic burden of SCD, β-thalassaemia and MDS.

METHODS: Searches were conducted in PubMed (including MEDLINE) and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) database. Studies that were not available in English were excluded, and no date limit was applied.

RESULTS: Searches returned 96 results, of which 30 were ultimately included. Thirteen studies were available as full-text studies (two of which were literature reviews), and 17 studies were ISPOR abstracts. Of the full-text studies, three focused on MDS, four on β-thalassaemia, four on SCD, one on SCD and β-thalassaemia, and one on β-thalassaemia, MDS and SCD. Only four full-text studies investigated the impact on indirect costs, with three of these studies focusing on β-thalassaemia, and one focusing on SCD. Results confirmed the significant burden of all three conditions with respect to medication, inpatient and outpatient costs, with individuals who require regular transfusions reporting higher medical costs. β-thalassaemia, MDS and SCD patients experienced absenteeism, presenteeism, and significant life changes impacting HRQoL. Results from ISPOR abstracts were aligned with those from the full-text studies.

CONCLUSIONS: β-thalassaemia, MDS and SCD pose a significant economic burden to healthcare systems, with transfusions and iron chelation therapies forming a significant proportion of these costs. Future studies could further explore the impact of specific treatments on the economic burden of β-thalassaemia, MDS and SCD.