Cost-effectiveness and cost–benefit analysis of the thalassaemia prevention programme in Guangdong, southern China

Objective This study aimed to evaluate the cost-effectiveness and economic benefits of the Guangdong thalassaemia prevention programme (GDTPP) to provide evidence for optimizing the prevention of thalassaemia. Methods We used the archived data of 2,987,437 couples of GDTPP in China between 2015 and 2024, and calculated prevention costs and therapy costs under blood transfusion and iron-chelation (BTIC) and hematopoietic stem cell transplantation (HSCT) for β- thalassaemia major (TM). Sensitivity analyses were conducted. Results Among all participation, 11,997 (0.40%) couples were identified as both carrying β 0 or α 0 -thalassaemia mutations. Prenatal diagnosis identified 3976 α-TM and 1566 β-TM affected fetuses. Assuming treatment under BTIC or HSCT, the total prevention cost was 498,267,571 RMB or 430,862,571 RMB, which was lower than the expected postpartum therapy costs of 1,631,851,866 RMB or 616,888,116 RMB. Sensitivity analysis showed that when the proportion of β-TM terminations exceeded 23.90% (BTIC) or 63.20% (HSCT), the total prevention cost was lower than the expected treatment cost. Cost-effectiveness also varied across regions. Conclusions The GDTPP is cost-effective and has achieved favorable outcomes. Curative treatments for TM can reduce prevention costs and improve quality of life, and they have the potential to reshape future thalassaemia prenatal prevention strategies in China.