Economic burden of adult patients with β-thalassaemia major in mainland China

• Published in: Orphanet journal of rare diseases 2023-08, Vol.18 (1), p.1-252, Article 252
• Main Authors: Zhen, Xuemei, Ming, Jing, Zhang, Runqi, Zhang, Shuo, Xie, Jing, Liu, Baoguo, Wang, Zijing, Sun, Xiaojie, Shi, Lizheng
• Format: Article
• Language: English
• Subjects: Adult patient; Adults; Age; American dollar; Blood; Blood diseases; Blood transfusion; Blood transfusions; Body weight; Caregivers; Chelation; Chelation therapy; China; Clinical practice guidelines; Coronaviruses; Costs; COVID-19; Demography; Economic burden; Ethnicity; Health care expenditures; Hemoglobin; Iron; Lifetime cost; Marital status; Medical research; Patients; Pediatrics; Provinces; Quality control; Questionnaires; Rare diseases; Thalassemia; β-thalassaemia major
• ISSN: 1750-1172; 1750-1172
• DOI: 10.1186/s13023-023-02858-4

Backgroundβ-thalassaemia major poses a substantial economic burden, especially in adults. We aimed to estimate the economic burden of adult patients with β-thalassaemia major from a societal perspective using the real-world data. According to the clinical guideline, we also estimated the annual medical costs for patients with the same body weight and calculated the lifetime medical costs over 50 years in mainland China.MethodsThis was a retrospective cross-sectional study. An online survey with snowball sampling covering seven provinces was conducted. We extracted patient demographics, caregiver demographics, disease and therapy information, caring burden, and costs for adult patients diagnosed with β-thalassaemia major and their primary caregivers. In the real world, we estimated the annual direct medical cost, direct nonmedical cost, and indirect cost. In addition, we calculated the annual direct medical cost and lifetime direct medical cost by weight with discounted and undiscounted rates according to the clinical guideline.ResultsDirect medical costs was the main driver of total cost, with blood transfusion and iron chelation therapy as the most expensive components of direct medical cost. In addition, adult patients with β-thalassaemia major weighing 56 kg were associated with an increase of $2,764 in the annual direct medical cost using the real-world data. The undiscounted and discounted (5% discount rate) total lifetime treatment costs were $518,871 and $163,441, respectively.ConclusionsPatients with β-thalassaemia major often encounter a substantial economic burden in mainland China. Efforts must be made to help policymakers develop effective strategies to reduce the burden and pevalence of thalassaemia.