OBJECTIVES: Beta-thalassemias are a group of genetic hemoglobinopathies which are caused by reduced or absent synthesis of the beta-chains of hemoglobin. Beta-thalassemia major, the most severe manifestation of disease, is also known as transfusion dependent thalassemia because patients require chronic transfusion to treat severe, symptomatic anemia. This study aimed to describe the current evidence of the economic burden related to beta-thalassemia, and to identify key factors correlated with an increased cost burden.

METHODS: Systematic searches were performed in databases such as MEDLINE, EMBASE and EconLit to identify studies assessing the economic burden of beta-thalassemia to patients, caregivers, healthcare systems, and society. Hand searches of INAHTA, NICE, CADTH, EUnetHTA, DARE, NHS EED and relevant conferences were conducted to identify further evidence. No geographic restrictions were applied and all studies published in English in the past 10 years were considered.

RESULTS: Of 345 references identified in the database search, 18 full-text publications met the inclusion criteria. Another two conference abstracts were included leading to an inclusion of 20 unique studies: 12 full economic evaluations, two observational studies, three cost-of-illness analyses, and three literature reviews. The 17 identified economic evaluations were conducted in the United Kingdom (5), Italy (3), Iran (2), and Thailand (2), as well as the United States (1), Korea (1), Israel (1), Egypt (1), and China (1). Direct medical costs represent the largest component of the total cost of beta-thalassemia therapy. Treatment costs represented a significant proportion of direct costs, specifically transfusion and chelation drug costs. Studies detailing direct non-medical costs and indirect costs are limited.

CONCLUSIONS: Overall, there is limited information on the economic burden of beta-thalassemia. Direct medical costs represent the major driver of total economic burden. The two key drivers of direct medical costs were blood transfusion and iron chelation therapy.