Iron chelation therapy in beta-thalassemia major: a systematic review with meta-analysis / 中国药学杂志

ABSTRACT

OBJECTIVE: 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 < 0.0001], urinary iron excretion [SMD = 1.04, 95% CI(0.61, 1.47), P < 0.00001], 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.