OBJECTIVES: We aimed to estimate economic burden of adult patients with β-thalassemia major from a societal perspective using real-world data; according to the clinical guideline, we would estimate annual medical cost of patients with the same weight, and calculate lifetime medical cost over 50 years as well in mainland China.

METHODS: This is a retrospective cross-sectional study. We extracted patient’s demographics, caregiver’s demographics, disease and therapy information, caring burden, and costs from adult patients diagnosed with β-thalassemia major and their primary caregivers. In the real world, the annual direct medical cost comprised cost for annual blood transfusion therapy, annual cost for iron chelation therapy, and annual cost for adverse reaction therapy. The annual direct non-medical cost comprised of annual transportation cost, annual accommodation cost, annual meal and nutrition cost, and annual nursing cost. The annual indirect cost was measured by annual cost due to loss labor productivity of patients and caregivers. We also calculated the annual direct medical cost by weight and cumulative lifetime direct medical cost according to the clinical guideline.

RESULTS: Our study estimated that annual direct medical cost, direct non-medical cost, and indirect cost of per adult patient with β-thalassemia major with 56kg were $20,531, $3,872, and $6,093, respectively. Direct medical cost was the main driver of total cost, and it was also revealed that blood transfusion and iron chelation therapy were the most expensive components of direct medical cost. In addition, compared with adult patients with β-thalassemia major with 56kg using real-world data, those were associated with $4,210 increase in the annual direct medical cost estimated based on the clinical guideline. It was estimated that the undiscounted and discounted total lifetime treatment cost was $790,392, and $248,969 in this study, respectively.

CONCLUSIONS: Adult patients with β-thalassemia major often encountered substantial economic burden in mainland China.