地拉罗司-去铁胺序贯疗法治疗重型β-地中海贫血铁过载的研究  

Study of sequential treatment with Deferasirox-Deferrioxamine inβ-thalassemia Major with iron overload

作　　者：何丽红[1] 孔慕贤[1] 李猛[1] 陈爱贞[1] 余建洪[1] 杨冠英[1] 

机构地区：[1]佛山市第一人民医院儿科,广东佛山528000

出　　处：《华夏医学》2015年第6期45-48,共4页Acta Medicinae Sinica

基　　金：2013年佛山市医学类科技攻关项目资助(201308011)

摘　　要：目的:研究地拉罗司-去铁胺序贯疗法治疗重型β-地中海贫血(β-TM)铁过载的疗效和安全性。方法:将输血及伴有铁过载的β-TM患儿150例,随机分为3组,分别应用地拉罗司(Ⅰ组)、地拉罗司-去铁胺序贯疗法(Ⅱ组)、去铁胺(Ⅲ组)治疗,统计分析3组患儿的临床资料、治疗的依从性。观察期1年。结果:3组患儿输血总量无差异,治疗前后SF比较Ⅰ组、Ⅱ组均明显降低,治疗后Ⅰ组、Ⅱ组较Ⅲ组明显降低(P<0.01)。治疗前后骨龄延迟病例比较Ⅰ组、Ⅱ组明显减少,治疗后Ⅲ组较Ⅰ组、Ⅱ组明显增多(P<0.01)。Ⅰ组、Ⅱ组治疗后肝功能异常病例均较治疗前明显减少,治疗后肝功能异常病例Ⅱ组较Ⅰ组、Ⅲ组明显减少(P<0.05)。Ⅲ组依从性不良病例较Ⅰ组、Ⅱ组明显增多(P<0.01)。治疗后身高和体重增长Ⅰ组、Ⅱ组较Ⅲ组明显增加(P<0.01)。结论:地拉罗司-去铁胺序贯疗法治疗β-TM铁过载是安全有效的,可以作为治疗铁过载的方案之一。Objective：To study the effect and safety of sequential treatment with Deferasirox（DFX）-Deferrioxamine（DFO）inβ-thalassemia Major（β-TM）.Methods：150children withβ-TM who received regular blood transfusion and chelation were enrolled.They were divided into three groups randomly.The treatment were DFX（Ⅰgroup）or DFX-DFO（Ⅱgroup）or DFO（Ⅲgroup）.The clinical data and compliance were analyzed.The follow-up time was one year.Results：Amount of transfusion in three groups was not different.SF after treatment ofⅠgroup and Ⅱgroup had been lower obviously than that before treatment,SF after treatment inⅠgroup and Ⅱgroup were lower than that ofⅢ group obviously（P〈0.01）.The case of skeleton age delay inⅠgroup and Ⅱgroup had been lower obviously than that before treatment,The case of skeleton age delay after treatment inⅢ group had been higher obviously than that ofⅠgroup andⅡgroup（P〈0.01）.The cases of abnormal liver function after treatment had been reduced obviously than that before treatment in Ⅰgroup and Ⅱgroup,The case of abnormal liver function after treatment in Ⅱgroup had been lower thanⅠgroup andⅢgroup（P〈0.05）.Poor compliance had been higher obviously in Ⅲ group than that ofⅠgroup andⅡgroup（P〈0.01）.The increase value of height and weight after treatment had been obviously increased inⅠgroup andⅡgroup than that ofⅢgroup（P〈0.01）.Conclusions：The sequential treatment with DFX-DFO is effective and safety inβ-TM with iron overload.It can be one of the chelate methods in iron overload.

关 键 词：重型Β-地中海贫血 地拉罗司 去铁胺 血清铁蛋白 铁负荷 

分 类 号：R556.61[医药卫生—血液循环系统疾病]