目的 系统评价去铁胺与去铁酮治疗重型β-地中海贫血的临床疗效。方法 检索Cochrane图书馆、PubMed、EMBASE、中国期刊全文数据库、中国生物医学文献数据库和维普期刊数据库。以血清铁蛋白、肝组织铁浓度、尿铁排出量、心室射血分数值和心肌铁含量5个指标评价去铁胺、去铁酮和联合治疗的疗效，采用RevMan 5.0软件进行Meta分析。结果:纳入符合条件的9篇RCT文献，共654名患者。Meta分析结果显示，去铁胺与去铁酮联用在以下方面均优于单用二者之一，且差异有统计学意义：血清铁蛋白下降值[WMD=-215.37，95%CI(-395.35，-35.39)，P=0.02]、肝组织铁浓度下降值[SMD=-1.06，95%CI(-1.54，-0.58)，P<0.000 1]、尿铁排出量变化值[SMD=1.04，95%CI(0.61，1.47)，P<0.000 01]与心室射血分数值[WMD=3.37，95%CI(0.79，5.95)，P=0.01]；而在心肌铁含量变化方面差异无统计学意义[WMD=1.70，95%CI(-2.78，6.18),P=0.46]。去铁胺组与去铁酮组单独疗效中血清铁蛋白下降值[WMD=133.45，95%CI(-48.92315.82)，P=0.15]、心室射血分数值变化程度[WMD=0.96，95%CI(-2.74，4.66)，P=0.40]、心肌铁含量变化程度[WMD=1.50，95%CI(-1.70，4.70),P=0.36]均不具有统计学意义。结论 :本篇Meta分析结果提示，去铁胺与去铁酮联用疗效优于二者之一单用，从而为当前国内重型β-地中海贫血的治疗提供了一种新的治疗选择。但由于纳入研究样本量小且质量较低，上述结论尚需要高质量、大样本的双盲随机对照试验加以证实。

The efficacy and safety of deferrioxamine(deferoxamine,DFO)and deferiprone(DFP)has been widely reported and researched so far， some scholars have reported the efficacy of the combination of the two drugs， but it is still lack of adequate research to prove the efficacy between combination therapy and single administration This study is evaluate the efficacy of deferrioxamine， deferiprone and combination therapy in beta thalassemia major patientsMETHODS The randomized controlled trials(RCT)were collected from the Cochrane library，PubMed，EMBASE， CBM，CNKI and VIP，etc.Serum ferritin(SF)，liver iron concentration(LIC)，urinary iron excretion(UIE)， ejection fraction(EF)and myocardial iron concentration(MIC)were chosen as evaluation index to evaluate the efficacy. Studies were screened， data were extracted，and the methodological quality was assessed by two reviewers independently. Meta-analyses were conducted by using RevMan 5.0 software RESULTS Nine randomized controlled studies involving 654 patients were included The results showed that compared with the group of single administration of either deferiprone or deferoxamine， the experimental group of deferiprone combined with deferoxamine was superior in the following aspects with significant differences：Serum ferritin levels [WMD=-215.37，95%CI(-395.35， -35.39)，P=0.02]，liver iron concentration[SMD=-1.06，95%CI(-1.54，-0.58)，P<0.000 1]，urinary iron excretion [SMD=1.04，95%CI(0.61，1.47)，P<0.000 01]，ejection fraction[WMD=3.37，95%CI(0.79， 5.95)，P=0.01]. But no statistically significant variation in myocardial iron concentration between deferoxamine combined with deferiprone and monotherapy [WMD=1.70，95%CI(-2.78，6.18)，P=0.46] There was no statistically significant variation in serum ferritin[WMD=13345，95%CI(-48.92，315.82)，P=0.15]， ejection fraction [WMD=0.96，95%CI(-2.74，4.66)，P=0.40]，myocardial iron concentration [WMD=1.50，95%CI(-1.70， 4.70 )，P=0.36] during deferoxamine versus deferiprone treatment CONCLUSION According to the domestic evidence， deferoxamine combined with deferiprone for treating betathalassemia major is superior to deferoxamine or deferiprone monotherapyIt provides a new and prospective therapeutic method for betathalassemia major However，for the quality restrictions of the included studies， this conclusion has to be further verified by high quality， large scale and double blinded randomized controlled trials.