地拉罗司治疗重型β-地中海贫血铁过载患儿临床疗效及安全性研究  

Curative effects and safety of deferasirox in treatment of iron overload in children with β-thalassemia major

作　　者：高红英[1] 李其[1] 陈娟娟[1] 陈光福[1] 李长钢[2] 

机构地区：[1]深圳市第二人民医院儿科,广东深圳518035 [2]深圳市儿童医院血液肿瘤科,广东深圳518026

出　　处：《中国当代儿科杂志》2011年第7期531-534,共4页Chinese Journal of Contemporary Pediatrics

基　　金：深圳市科技计划项目(No.200903051);深圳市科技计划项目(重点)(No.201001019)

摘　　要：目的探讨铁螯合剂地拉罗司(deferasirox,DFX)治疗重型β-地中海贫血(β-thalassemia major,β-TM)铁过载患儿的疗效及安全性。方法随机选择24例规律输血的β-TM铁过载患儿,参加DFX不同服药剂量的临床研究,调查血清铁蛋白(SF)的变化及不良反应。并将持续服用DFX 5年患儿与同期使用去铁胺联合去铁酮治疗患儿(对照组)的心脏MRI T2*、肝脏MRI T2*值进行比较。结果 DFX每日20～30 mg/kg的起始剂量对于铁过载患儿无明显效果,加量至每日30～40 mg/kg后SF水平下降显著(U=58,P<0.01);不良反应以血清肝脏转氨酶升高最为常见,其次为血清肌酐非进行性升高。持续DFX治疗5年组SF水平明显低于对照组(1748±481 ng/mL vs 3462±1744 ng/mL,P<0.05);肝脏MRI T2*值明显高于对照组(8.5±2.9 ms vs 2.7±1.9 ms,P<0.01)。两组心脏MRI T2*均值比较差异无统计学意义。结论 DFX能显著降低β-TM患儿SF水平,并显示出剂量依赖性变化;其对心脏铁负荷的减少未显示出明显优势,而对肝脏铁负荷的减低疗效显著。DFX治疗的不良反应以肝酶升高、血清肌酐非进行性升高为主。Objective To study the effectiveness and safety of deferasirox（DFX） in the treatment of iron overload in children with β-thalassemia major.Methods Twenty-four β-thalassemia major children with iron overload who received regular blood transfusion were randomly enrolled.The serum feritin（SF） levels were measured in the patients after different doses of DFX treatment.The DFX treatment-related adverse events were observed.The values of cardiac MRI T2＊ and liver MRI T2＊ were compared between the patients receiving DFX treatment for 5 years and the patients treated with deferoxamine and deferiprone.Results The patients with iron overload did not respond to DFX at the initial dose of 20-30 mg/kg·d.However,the SF level decreased significantly after the dose of DFX increased to 30-40 mg/kg·d（U=58,P〈0.01）.Serum liver transaminase elevation was the most common adverse effect,followed by non-progressive elevation in serum creatinine level.The mean SF level was significantly lower（1748±481 ng/mL vs 3462±1744 ng/mL;P〈0.05）,in contrast,the liver MRI T2＊ value was significantly higher（8.5±2.9 ms vs 2.7±1.9 ms;P〈0.01） in patients receiving DFX treatment for 5 years than in the controls.There were no significant differences in the cardiac MRI T2＊ value between the two groups.Conclusions DFX can reduce SF levels in a dose-dependent manner in children with β-thalassemia major.It can significantly lower liver iron overload but not cardiac overload.Serum liver transaminase elevation and non-progressive elevation in serum creatinine level are major adverse effects in DFX treatment.

关 键 词：重型Β-地中海贫血 地拉罗司 血清铁蛋白 铁负荷 儿童 

分 类 号：R725.5[医药卫生—儿科]