不同铁螯合剂治疗重型β地中海贫血患者疗效观察

Clinical effect of iron chelatorto in the treatment of patients with thalassemia major

目的探讨不同铁螯合剂在治疗重型β地中海贫血患者铁负荷的临床疗效。方法收集既往两家医院诊断治疗并完成随访的重型β地中海贫血铁过载患者49例,根据使用不同祛铁治疗方案分为三组,A组18例,B组21例,C组10例,A组患者采用足疗程联合祛铁治疗方案:去铁胺(DFO)30~50 mg/(kg·d),每周使用≥5 d;去铁铜(DFP) 75 mg/(kg·d),每周使用7 d。B组患者采用不足疗程联合祛铁治疗方案:DFO 30~50 mg/(kg·d),每周使用2~5 d;DFP 75 mg/(kg·d),每周使用3~7 d。C组单用祛铁治疗方案:地拉罗司(DFX)20~30 mg/(kg·d),每周使用7 d。疗程均为一年。三组患者治疗前后都经MRI T2~*技术检测心脏及肝脏铁过载情况,对比分析治疗前后三组患者血清铁蛋白(SF)值、心脏MRI T2~*值、肝脏MRI T2~*值的变化。结果患者治疗1年后,三组的SF值与治疗前比较均有下降(P<0.01),治疗后三组SF值比较,差异有统计学意义(F=4.310,P<0.05),A组的SF较其余两组下降更明显(P<0.05);治疗1年后,心脏MRI T2~*值与治疗前比较均显著升高(P<0.05),治疗后三组心脏MRI T2~*值比较,差异有统计学意义(F=340.135,P<0.01),A组的心脏MRI T2~*值较其余两组升高更明显(P<0.01)。治疗1年后肝脏MRI T2~*值与治疗前相比均显著升高(P<0.01),治疗后三组患者肝脏MRI T2~*值比较差异无统计学意义(F=0.118,P>0.05)。消化道反应、骨关节疼痛及粒细胞减少是最常见的不良反应。结论足疗程或不足疗程联合祛铁治疗及地拉罗司均可以有效缓解重型β地中海贫血患者铁负荷,其中以足疗程联合DFO+DFP治疗地中海贫血患者在降低SF值、心脏铁过载效果更优,不良反应轻微,常见消化道反应及骨关节疼痛,值得在临床中推广。

Objective Our study aimed to compare the effectiveness of iron chelator on serum ferritin(SF),heart and liver iron function in patients with thalassemia major.Methods We collected the data of 49 patients with thalassemia major,who were highly iron overloaded and previously treated and followed up by our hospital and the First Affiliated Hospital of Guangxi Medical University,of which 21 cases were in the group with insufficient treatment,18 cases were in the group with sufficient treatment,and 10 cases were in the group treated with deferasirox.Cardiac and liver iron overload were detected by MRI T2~* technology before and after treatment in the three groups of patients.Changes in SFvalue,cardiac MRI T2~* value,and liver MRI T2~* value were compared and analyzed.Results After one years of treatment,the serum SF value of the patients and the heart and liver MRI T2~* values were better than before treatment(P<0.05).The difference in serum SF between the three groups were statistically significant(F=4.310,P<0.05);There was no significant difference between the groups in the T2~* values of liver MRI(F=0.118,P>0.05).Between the three groups,heart MRI was significantly different(F=40.135,P<0.01).There were 18 cases of adverse reactions;9 were in the group with sufficient treatment,2 were in the group with insufficient treatment,and 2 cases were in the talazol group.Conclusion Deferoxamine,deferiprone and deferasirox can effectively alleviate the condition of patients with thalassemia major,who were highly iron overloaded.Among them,thecombination of desferrioxamine and deferasirox with sufficient duration of treatment is more effective in reducing cardiac iron overload,and is worthy of clinical promotion.