Pharmacoeconomic studies of transfusion-dependent β thalassemia：systematic review
- VernacularTitle:输血依赖型β地中海贫血药物经济学研究的系统评价
- Author:
Junni DU
1
;
Yan LI
1
;
Pingyu CHEN
1
,
2
;
Aixia MA
1
Author Information
1. School of International Pharmaceutical Business，China Pharmaceutical University，Nanjing 211198，China
2. Evaluation and Research Center of Pharmacoeconomics，China Pharmaceutical University，Nanjing 211198，China
- Publication Type:Journal Article
- Keywords:
transfusion-dependent β thalassemia;
pharmacoeconomics;
systematic review;
blood transfusion and iron chelation
- From:
China Pharmacy
2025;36(19):2415-2421
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVE To systematically evaluate pharmacoeconomic studies on treatment strategies for transfusion- dependent β thalassemia （TDT） both in China and internationally， to inform clinical decision-making and health resource allocation， and provide methodological references for future domestic research in China. METHODS Databases including PubMed， Embase， Web of Science， the Cochrane Library， CNKI， Wanfang Data， and VIP were searched for pharmacoeconomic studies related to TDT treatments from inception to December 2024. Two researchers independently screened the literature， extracted data， and assessed risk of bias using the Consolidated Health Economic Evaluation Reporting Standards 2022 （CHEERS 2022） checklist. A qualitative systematic review was conducted focusing on model structure， costs， health outcomes， uncertainty analysis， and economic evaluation results. RESULTS A total of 1 685 articles were initially identified， and 13 pharmacoeconomic studies were ultimately included. The overall quality of the included studies was relatively high， covering interventions including blood transfusion and iron chelation therapy （BT-ICT）， hematopoietic stem cell transplantation （HSCT）， and gene therapy. All studies employed cost-utility analysis from societal， healthcare system， and payer perspectives. Markov models were most frequently used in model design. Most studies adopted a lifetime horizon with a cycle length of one year and a discount rate of 1.5% to 5%. Costs mainly focused on direct medical costs， sourced from national insurance databases and published literature. Utility values were primarily derived from literature or measured using techniques such as time trade-off. Most studies conducted both deterministic and probabilistic sensitivity analyses， identifying the costs of iron chelators and utility values as key influential parameters. Some studies also performed scenario analyses. CONCLUSIONS HSCT was likely more cost-effective than BT-ICT for pediatric/ adolescent patients. BT-ICT demonstrated superior cost-utility for adult patients， with the deferiprone regimen potentially being the most cost-effective option. Current domestic and international pharmacoeconomic evaluations for TDT remain limited. Future research should adopt a societal perspective， use a lifetime horizon， and strictly follow the CHEERS 2022 checklist when conducting pharmacoeconomic evaluations of treatments for TDT.
