广西医学2025，Vol.47Issue(4)：578-584,7.DOI:10.11675/j.issn.0253-4304.2025.04.15
羟基脲治疗输血依赖型β-地中海贫血的临床效果及药物经济学评价
Clinical effect and pharmacoeconomic evaluation of hydroxyurea for the treatment of transfusion-dependent β-thalassemia
摘要
Abstract
Objective To evaluate the effectiveness,safety,and economy of hydroxyurea for the treatment of transfusion-dependent β-thalassemia.Methods A retrospective study was conducted involving 80 patients with transfusion-dependent β-thalassemia as the research subjects,therein the hydroxyurea group(40 cases)received conventional blood transfusion combined with hydroxyurea for treatment,while the conventional treatment group(40 cases)received conventional blood transfusion for treatment.The efficacy and safety were compared between patients of the two groups.A Markov model was constructed for cost-utility analysis,and economy was compared between the two groups.Results After treatment,the hemoglobin level in the hydroxyurea group was higher than that before treatment and in the conventional treatment group(P<0.05).However,no statistically significant difference was observed between the two groups in terms of total effective rate of treatment,transfusion frequency,transfusion volume(P>0.05),and safety was similar.The results of cost-utility analysis revealed that compared with the conventional treatment group,the hydroxyurea group had an incremental cost of 1559.13 RMB per patient and an incremental utility of 0.20 quality adjusted life year(QALY),resulting in an incremental cost effectiveness ratio of 7522.47 RMB/QALY.The results indicated that hydroxyurea demonstrated cost-utility advantage to a certain extent when the willingness-to-pay value exceeded 7522.4 RMB.Probabilistic sensitivity analysis confirmed the stability of the findings of cost-utility analysis.Conclusion For patients with transfusion-dependent β-thalassemia,the addition of hydroxyurea to conventional blood transfusion therapy offers clinical and cost-utility advantages compared with conventional treatment regimen to a certain extent.关键词
β-地中海贫血/输血依赖型/羟基脲/输血治疗/Markov模型/疗效/安全性/成本-效用分析Key words
β-Thalassemia/Transfusion-dependent type/Hydroxyurea/Blood transfusion therapy/Markov model/Efficacy/Safety/Cost-utility analysis分类
药学引用本文复制引用
蒋慧娴,黄天敏,郭雁翔,颜潇旖,唐甘翎,李静怡,黄肖曼,黄振光,张宏亮..羟基脲治疗输血依赖型β-地中海贫血的临床效果及药物经济学评价[J].广西医学,2025,47(4):578-584,7.基金项目
自治区卫生健康委药品临床综合评价课题(YPPJ004) （YPPJ004）
2022白求恩·求索-药学科研能力建设项目(Z04JKM2021005) （Z04JKM2021005）
