去铁胺联合去铁酮在重症地中海贫血患者中的疗效及对铁代谢状态的影响研究

卜声镝+陈江明+菜宁

【摘要】 目的：探讨重症地中海贫血患者应用去铁胺与去铁酮联合治疗的临床效果及对患者铁代谢状态的影响研究。方法：选取2014年3月-2016年5月在医院接受诊治的重症地中海贫血患者90例为研究对象，根据治疗方案不同分两组，其中对照组患者43例采用常规去铁胺治疗，观察组患者47例采用去铁胺联合去铁酮治疗，以连续治疗12个月为观察期，比较两组患者在治疗后3、6、12个月的尿铁排泄（UIE）情况，血清铁蛋白（SF）、肝内铁浓度（LIC）及促红细胞生长素（EPO）等体内铁代谢状态水平的变化，同时分析两组患者的治疗总有效率。结果：两组患者在治疗3、6、12个月后的UIE排泄量均逐渐增多，且观察组明显多于对照组（P<0.05）；观察组患者治疗后的SF、LIC、EPO水平均明显低于对照组（P<0.05）；观察组患者的治疗有效率为93.62%，明显高于对照组79.07%（P<0.05）。结论：重症地中海贫血患者采用去铁胺和去铁酮联合治疗，能够有效减少机体内铁过载状态，增加铁尿排泄量，具有显著的临床治疗效果。

【关键词】 去铁胺； 去铁酮； 重症地中海贫血； 铁代谢状态； 疗效

【Abstract】 Objective：To investigate the clinical effect of Deferoxamine combined with Deferiprone in patients with severe thalassemia and its effect on iron metabolism in patients.Method：90 patients with severe thalassemia in the hospital from March 2014 to May 2017 were selected.According to the treatment program，they were divided into two groups.43 patients in control group were treated with Deferoxamine，and 47 patients in observation group were treated with Deferoxamine combined with Deferiprone.Continuous treatment for 12 months was observation period.The levels of urinary iron excretion（UIE） in two groups were compared at 3，6 and 12 months after treatment，the levels of iron metabolism in serum ferritin（SF），intrahepatic iron concentration（LIC） and erythropoietin（EPO） of two groups were compared，the total effective rates of two groups were also analyzed.Result：After treatment for 3，6 and 12 months，the UIE excretion of two groups increased gradually，and observation group was significantly more than that of control group（P<0.05）.After treatment，the levels of SF，LIC，and EPO in observation group were significantly lower than those of control group（P<0.05）.The effective rate of treatment in observation group was 93.62%，significantly higher than that of control group of 79.07%（P<0.05）.Conclusion：Deferoxamine combined with Deferiprone in the treatment of severe thalassemia，can effectively reduce the iron overload，increase the urinary excretion，and have significant clinical therapeutic effect.

【Key words】 Deferoxamine； Deferiprone； Severe thalassemia； Iron metabolism； Efficacy

First-authors address：Gongren Hospital of Wuzhou City，Wuzhou 543001，China

doi：10.3969/j.issn.1674-4985.2017.31.010

地中海貧血是一种由于染色体基因缺陷导致珠蛋白链合成障碍的溶血性贫血，此疾病的种类繁多且临床表现轻重不一，多数患者为低色素小细胞贫血[1]。据报道，这种贫血疾病主要高发在热带地区，因此得名地中海贫血[2-3]。我国的发病人群主要集中海南、广西、广东等地区，其发病率约18.7%[4]。研究调查，在每100人中就有5～6例携带这种缺陷基因，对人类生命健康产生严重威胁，尤其是重症患者的视力和听力都有严重影响，逐年升高的致残率和死亡率都不容忽视[5]。临床研究表明，机体内铁离子的蓄积是导致患者肝肾等重要器官损伤的重要原因，应用去铁胺是抑制铁过载而发生并发症的有效治疗方式[6-7]，然而由于患者存在个体差异，目前对于去铁胺对机体产生的变态反应和毒副作用并没有安全的控制措施，许多患者无法承担这样的治疗风险[8]。……