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输血依赖型β地中海贫血药物经济学研究的系统评价
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| 篇名： | 输血依赖型β地中海贫血药物经济学研究的系统评价 |
| TITLE： | Pharmacoeconomic studies of transfusion-dependent β thalassemia：systematic review |
| 摘要： | 目的 系统评价国内外输血依赖型β地中海贫血（TDT）治疗方案的药物经济学研究，以期为优化临床决策与卫生资源配置提供依据，并为我国开展后续相关研究提供方法学参考。方法采用计算机检索PubMed、Embase、WebofScience、theCochraneLibrary、中国知网、万方数据库和维普网等数据库，搜集TDT相关药物经济学研究，检索时限为建库起至2024年12月。由2名研究者独立筛选文献、提取资料并采用《卫生经济学评价报告标准共识2022》（CHEERS2022）清单进行偏倚风险评价后，从模型结构、成本与健康产出、不确定性分析及经济性评价结果等方面进行定性系统评价。结果初检获得1685篇文献，最终纳入13个基于模型的药物经济学研究；纳入研究总体质量较高；干预措施包含输血联合铁螯合疗法（BT-ICT）、造血干细胞移植（HSCT）和基因疗法。所有研究均采用成本-效用分析评价方法，研究角度涉及全社会、卫生体系和医保支付方。模型设定方面，马尔可夫模型应用最多；多数研究的研究时限为终身，模型周期为1年，贴现率为1.5%～5%。纳入成本主要考虑直接医疗成本，数据来源于国家医保数据库和已发表文献等；效用值主要来源于文献或通过时间权衡法等测量得到。多数研究同时采用确定型敏感性分析与概率敏感性分析，铁螯合剂价格与效用值等参数对经济性结果影响较大；部分研究采用了情境分析。结论HSCT相较于BT-ICT在儿童/青少年患者中可能更具经济性；BT-ICT对成年患者更具成本-效用优势，其中去铁酮方案的经济性可能最好。现有的国内外TDT经济性评价研究数量较少；建议未来研究者基于全社会视角，采用全生命周期时限，严格遵循CHEERS2022清单要求开展TDT的药物经济学评价。 |
| 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. |
| 期刊： | 2025年第36卷第19期 |
| 作者： | 杜珺妮；李艳；陈平钰；马爱霞 |
| AUTHORS： | DU Junni，LI Yan，CHEN Pingyu，MA Aixia |
| 关键字： | 输血依赖型β地中海贫血；药物经济学；系统评价；输血联合铁螯合疗法；造血干细胞移植；基因疗法 |
| KEYWORDS： | transfusion-dependent β thalassemia； pharmacoeconomics； systematic review； blood transfusion and iron chelation |
| 阅读数： | 5 次 |
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