目的：研究去铁胺（ DFO）治疗重型β－地中海贫血患儿持续每天使用的祛铁效果及不良反应。方法2014年1月至2015年1月于广东省惠州市第一人民医院住院治疗的70例重型β－地中海贫血患者，根据随机数表法分为试验组35例，对照组35例。当血清铁蛋白（ SF）＞1000μg／L时开始祛铁，所有患儿均给予DFO治疗，实验组患儿每日持续使用去铁胺，对照组每隔1天使用1次去铁胺。对于患者可能出现的眼部不良反应进行评估及眼科专科检查。连续治疗12个月，每12周进行一次血清血蛋白检查、去铁胺剂量的调整及视力评估。比较给药前后的铁负荷变化及眼部不良反应情况。结果治疗前两组SF、尿铁排泄量（UIE）水平差异均无统计学意义（P＞0．05）；两组治疗3个月、6个月、9个月及12个月的SF相比，试验组明显低于对照组（t值分别为4．357、6．183、5．146、6．002，均P＜0．05）；UIE相比，试验组明显高于对照组，且差异均具有统计学意义（t值分别为3．713、4．262、6．113、4．002，均P＜0．05）。不良反应情况：治疗组治疗6个月出现3例视力模糊，治疗后12个月其中1例颜色视力受损（色觉障碍），1例视野缺损伴有盲点，1例角膜浊斑，另有5例出现视力下降，无视力丧失情况；不良反应率达22．9％；对照组治疗12个月出现2例视力下降，不良反应率为8．6％，两组眼睛不良反应差异具有统计学意义（χ2＝4．20，P＜0．05）。结论去铁胺对重型β－地中海贫血患儿的持续治疗优于间歇治疗，并且有显著的祛铁效果，其眼部不良反应较高，需要得到临床医生的注意，适时适当服药或停药，改善患者的生存质量。%Objective To study the effect of removing iron and adverse reactions of Deferoxamine ( DFO) in the treatment of severe beta-thalassemia major (β-TM) in children.Methods Seventy cases of severeβ-TM treated in the First People’s Hospital of Huizhou during the period of January 2014 to January 2015 were randomly divided into experimental group (35 cases) and control group (35 cases).When SF>1 000ug/L removing iron was performed , and all the children were given DFO .In the experimental group continuous use of DFO was provided daily , while the control group used DFO every other day .Adverse reactions possibly occurring in eyes were assessed and specialist examinations were performed in department of ophthalmology .Treatment lasted 12 months.Serum protein levels were examined , dosage of DFO was adjusted and visual acuity was assessed every 12 weeks.Iron load and adverse reactions in eyes were compared before and after administration.Results There were no significant differences in levels of UIE and SE before treatment between two groups ( P>0.05). After treatment for 3 months, 6 months, 9 months and 12 months, SF was significantly lower in the experimental group ( t value was 4.357, 6.183, 5.146 and 6.002, respectively, all P<0.05), while UIE level was significantly higher in the group with significant difference (t value was 3.713, 4.262, 6.113 and 4.002, respectively, all P<0.05).In the experimental group there were 3 cases of blurred vision after 6 months of treatment .After 12 months of treatment there was 1 case of color vision defect ( color deficiency ) , 1 case of visual field defect associated with blind spots , 1 case of corneal opacities , 5 cases of poor vision , but no case with loss of vision . Adverse reaction rate was 22.9% in the experimental group .In the control group 12 months after treatment there were 2 cases of poor vision loss, and the adverse reaction rate was 8.6%.The difference in adverse reactions in eyes was statistically significant between two groups (χ2 =4.20,P<0.05).Conclusion The continuous treatment of severe β-TM in children is better than the intermittent treatment , and it has significant effect of removing iron .The incidence of adverse reaction in eyes is relatively high , which needs to attract attention of clinicians , timely appropriate medication or stop drug so as to improve the quality of life of patients .
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