中文摘要：

目的：比较地拉罗司（DFX）及去铁胺（DFO）治疗输血依赖型地中海贫血（TDT）的效果,评价单药治疗的优势及局限性。方法：回顾分析我院门诊2013年1月-2015年6月TDT合并铁过载并接受DFX或DFO治疗的病例资料。DFX组58例患儿选择DFX 20~35 mg/（kg·d）治疗,DFO组27例患儿选择DFO 25~45 mg/（kg·d）治疗,每周5 d。自用药起随访1年,每3~6个月检测血清铁蛋白（SF）水平、肝肾功能、血常规。结果：两组患儿中位年龄、平均铁摄入率及治疗前SF比较差异无统计学意义。随访6个月,DFX组和DFO组SF下降均值分别为168（-2 650,7 254）ng/mL和170（-260,599）ng/mL（P〉0.05）。DFX组SF下降与剂量呈正相关（P〈0.05）。随访7~12个月,DFX组与DFO组SF下降均值分别为212（-370,795）ng/mL和-1 330（-2 454,-206）ng/mL（P〈0.05）。结论：DFX较DFO治疗TDT有优势,宜使用耐受范围内最大剂量。DFX可稳定SF,宜长期用药。DFO安全剂量用于大量输血患儿效果不佳。

英文摘要：

Objective：To study the effects of deferasirox（DFX） and deferoxamine（DFO） for iron overload in transfusion dependent thalassemia（TDT）.Methods：The children of TDT with iron overload accepted either DFX or DFO monotherapy between January 2013 and June 2015 were study retrospectively.Fifty-eight children in DFX group were given DFX,20 ~ 35 mg/（kg·d） treatment,twentyseven children in DFO group were given DFO,25~45 mg/（kg·d） treatment,every 5 days a week.The followed up was carried out for one year since medication.The level of serum ferritin（SF）,liver and kidney function,blood routine examination were detected every 3~6 months.Results：The median age,averages of iron intake,and SF before therapy were not different significantly in two groups.The zere to six months follow-up showed that mean SF decreased by 168（-2,650,7,254） ng/mL and 170（-260,599） ng/mL（P〉0.05）in the DFX group and the DFO group,respectively.The amount of decreased SF was positively related to dose in group DFX（P〈0.05）.The seven to twelve months follow-up showed that mean SF decreased by 212（-370,795） ng/mL and -1,330（-2,454,-206） ng/mL（P〈0.05） in the DFX group and the DFO group,respectively.Conclusion：DFX is more competitive than DFO.The effect of DFX is positively related to dose which should not be limited until it is beyond the tolerance.DFX is capable of stabilizing the concentration of SF,meaning persistent use needed.DFO monotherapy is not favorable for high dose transfusion children.