Xia Sujian, Zhang Weidong, Huang Liting, Jiang Hong
Division of Medical Statistics, School of Medicine, University of Jinan, Guangzhou City, Guangdong, China.
Novartis Pharmaceuticals Oncology, Beijing, China.
PLoS One. 2013 Dec 23;8(12):e82662. doi: 10.1371/journal.pone.0082662. eCollection 2013.
A meta-analysis was conducted to investigate the efficacy and safety of three main iron chelators, namely, deferoxamine (DFO), deferiprone (DFP) and deferasirox (DFX) for thalassemia major (TM) patients.
Randomized controlled trials comparing mono-therapy DFO, DFP, DFX and combined DFP with DFO therapy in TM patients from January 1990 to December 2012 were searched and selected. Two independent authors assessed data from extracted randomized trials for efficacy and safety in the measurements of serum ferritin (SF), live iron concentration (LIC), myocardial iron content (MIC), left ventricular ejection fraction (LVEF) and adverse events (AEs).
Sixteen studies were selected. In the comparison of DFP versus DFO treatment groups, a significant difference was revealed on MIC and LVEF (P=0.01 and P=0.007, respectively) but not on SF or LIC level (P=0.65 and P=0.37, respectively). In comparing combined therapy (DFP plus DFO) versus DFO, a significant difference was shown on MIC and LVEF measurements (P<0.00001 and P=0.003, respectively), but not on SF or LIC levels (P=0.93 and P=0.62, respectively). Moreover, the combined DFP with DFO treatment had significantly higher risk than DFO treatment (RR 1.46 with 95%CI 1.04 to 2.04). When comparing DFX with DFO, a significant difference was shown on the SF level (P=0.003), and there was no difference between DFX and DFO in safety evaluation (RR 1.53 with 95%CI 0.31 to 7.49).
Findings indicated that the most effective and safe iron chelators remains to be proven, and further large-scale, long-term studies are needed.
进行一项荟萃分析，以研究三种主要铁螯合剂，即去铁胺（DFO）、去铁酮（DFP）和地拉罗司（DFX）对重型地中海贫血（TM）患者的疗效和安全性。
检索并选取1990年1月至2012年12月期间比较DFO、DFP、DFX单药治疗以及DFP与DFO联合治疗TM患者的随机对照试验。两位独立作者评估提取的随机试验数据，以分析血清铁蛋白（SF）、肝脏铁浓度（LIC）、心肌铁含量（MIC）、左心室射血分数（LVEF）测量值以及不良事件（AE）方面的疗效和安全性。
选取了16项研究。在DFP与DFO治疗组的比较中，MIC和LVEF显示出显著差异（分别为P = 0.01和P = 0.007），但SF或LIC水平无显著差异（分别为P = 0.65和P = 0.37）。在联合治疗（DFP加DFO）与DFO的比较中，MIC和LVEF测量值显示出显著差异（分别为P < 0.00001和P = 0.003），但SF或LIC水平无显著差异（分别为P = 0.93和P = 0.62）。此外，DFP与DFO联合治疗的风险显著高于DFO治疗（RR 1.46，95%CI为1.04至2.04）。在DFX与DFO的比较中，SF水平显示出显著差异（P = 0.003），在安全性评估方面DFX与DFO无差异（RR 1.53，95%CI为0.31至7.49）。
研究结果表明，最有效和安全的铁螯合剂仍有待证实，需要进一步开展大规模、长期研究。
