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地拉罗司治疗地中海贫血铁过载的快速卫生技术评估
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| 篇名： | 地拉罗司治疗地中海贫血铁过载的快速卫生技术评估 |
| TITLE： | Deferasirox for the treatment of iron overload in thalassemia ：a rapid health technology assessment |
| 摘要： | 目的 采用快速卫生技术评估的方法评价地拉罗司治疗地中海贫血铁过载的有效性、安全性和经济性，为临床合理用药提供循证依据。方法计算机检索PubMed、Embase、CochraneLibrary、NHSEED、CADTH、中国知网、万方数据库等中英文数据库/网站，收集地拉罗司对比去铁胺/去铁酮治疗地中海贫血铁过载的卫生技术评估（HTA）、系统评价/Meta分析和药物经济学研究，检索时限均为建库/建站起至2021年6月。在筛选文献、提取资料的基础上，分别采用HTAchecklist、系统性评价方法学质量评估表、经济评价报告标准量表评价纳入HTA报告、系统评价/Meta分析和药物经济学研究的文献质量，并对有效性、安全性结果进行定量描述，对经济学评价结果进行定性描述。结果从1569篇文献中筛选纳入1篇HTA报告、5篇系统评价/Meta分析和5篇药物经济学研究。纳入HTA报告、系统评价/Meta分析、药物经济学研究的质量等级均较高。多数研究报道，30mg/（kg·d）地拉罗司降低血清铁蛋白水平、肝脏铁过载水平的效果优于去铁胺；地拉罗司组患者的不良反应多为轻度胃肠道刺激症状、皮肤瘙痒、关节痛、转氨酶升高等，一般不影响后续治疗；去铁胺组与地拉罗司组患者严重不良反应比较，差异无统计学意义[RR＝0.96，95％CI（0.85，1.08），P＝0.52]；与去铁胺比较，地拉罗司的成本-效益较高；而与去铁酮相比，地拉罗司不太可能具有成本-效益。结论地拉罗司治疗地中海贫血铁过载具有良好的有效性和安全性；与去铁胺相比，地拉罗司在伊朗、英国等国家有良好的经济学优势。 |
| ABSTRACT： | OBJECTIVE To eval uate the effectiveness ，safety and economy of deferasir ox for the treatment of iron overload in thalassemia with rapid health technology assessment ，and to provide evidence-based basis for rational clinical use. METHODS Retrieved from Chinese and English database/website as PubMed ，Embase，Cochrane Library ，NHS EED ，CADTH，CNKI and Wanfang database ，health technology assessment （HTA），systematic evaluation/meta-analysis and pharmacological studies about deferasirox versus deferoxamine/deferiprone for the treatment of iron overload in thalassemia were collected from the inception to June 2021. Based on literature screening and data extraction ，the quality of literature about HTA reports ，systematic evaluation/ Meta-analysis and pharmacoeconomic research were evaluated with HTA checklist ，A Measurement Tool to As sess Systematic Reviews，standard scale of economic evaluation report. The effectiveness and safety results were described quantitatively ，and the economic evaluation results were described qualitatively. RESULTS One HTA report ，five systematic evaluation/meta-analysis and five pharmacoeconomic studies were selected from 1 569 literature. Included HTA reports ， systematic evaluation/meta-analysis，pharmacoeconomic studies were high in quality. Most studies reported that 30 mg/（kg·d） deferasirox was E-mail：aydgs@126.com better than deferoxamine in reducing the levels of s erum ferritin and liver iron overload ；ADR induced by deferasirox were mainly gastrointestinal irritation symptoms ，skin itching ，joint pain，transaminase elevation ，etc.，which generally did not affect subsequent treatment. There was no statistical significance in severe ADR between deferoxamine group and deferasirox group [RR ＝0.96，95％CI（0.85，1.08），P＝0.52]. Compared with deferoxamine，deferasirox had higher cost-effectiveness ；but deferasirox was less likely to be cost-effective than deferiprone. CONCLUSIONS Deferasirox has good effectiveness and safety for iron overload in thalassemia ，and has good economic advantages in Britain and Iran ，compared with deferoxamine. |
| 期刊： | 2022年第33卷第01期 |
| 作者： | 王君萍，张梅，汪国玉，吴正宇，周鹏，张家佳，宁丽娟，高杉 |
| AUTHORS： | WANG Junping，ZHANG Mei，WANG Guoyu，WU Zhengyu，ZHOU Peng，ZHANG Jiajia，NING Lijuan，GAO Shan |
| 关键字： | 地拉罗司；地中海贫血铁过载；快速卫生技术评估 |
| KEYWORDS： | deferasirox；iron overload in thalassemia ；rapid health technology assessment |
| 阅读数： | 307 次 |
| 本月下载数： | 6 次 |
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