U.O.C. Ematologia II con Talassemia, A.O. V. Cervello, Palermo, Italy.
Blood Cells Mol Dis. 2011 Oct 15;47(3):166-75. doi: 10.1016/j.bcmd.2011.07.002. Epub 2011 Aug 16.
The effectiveness of deferoxamine (DFO), deferiprone (DFP), or deferasirox (DFX) in thalassemia major was assessed. Outcomes were reported as means±SD, mean differences with 95% CI, or standardized mean differences. Statistical heterogeneity was tested using χ2 (Q) and I2. Sources of bias and Grading of Recommendations Assessment, Development and Evaluation system (GRADE) were considered. Overall, 1520 patients were included. Only 7.4% of trials were free of bias. Overall measurements suggest low trial quality (GRADE). The meta-analysis suggests lower final liver iron concentrations during associated versus monotherapy treatment (p<0.0001), increases in serum ferritin levels during DFX 5, 10, and 20 mg/kg versus DFO-treated groups (p<0.00001, p<0.00001, and p=0.002, respectively), but no statistically significant difference during DFX 30 mg/kg versus DFO (p=0.70), no statistically significant variations in heart T2* signal during associated or sequential versus mono-therapy treatment (p=0.46 and p=0.14, respectively), increases in urinary iron excretion during associated or sequential versus monotherapy treatment (p=0.008 and p=0.02, respectively), and improved ejection fraction during associated or sequential versus monotherapy treatment (p=0.01 and p<0.00001, respectively). These findings do not support any specific chelation treatment. The literature shows risks of bias, and additional larger and longer trials are needed.
评估了去铁胺（DFO）、地拉罗司（DFX）或去铁酮（DFP）在重型地中海贫血中的疗效。结果以平均值±标准差、95%置信区间的平均值差异或标准化平均值差异报告。使用 χ2（Q）和 I2 测试统计异质性。考虑了偏倚来源和推荐评估、制定与评估系统（GRADE）。共有 1520 名患者入选。只有 7.4%的试验无偏倚。总体测量表明试验质量较低（GRADE）。荟萃分析表明，联合治疗与单一治疗相比，最终肝脏铁浓度较低（p<0.0001），DFX 5、10 和 20mg/kg 与 DFO 治疗组的血清铁蛋白水平升高（p<0.00001、p<0.00001 和 p=0.002），但 DFX 30mg/kg 与 DFO 之间无统计学差异（p=0.70），联合或序贯与单一治疗相比，心脏 T2*信号无统计学差异（p=0.46 和 p=0.14），联合或序贯与单一治疗相比，尿铁排泄增加（p=0.008 和 p=0.02），联合或序贯与单一治疗相比，射血分数改善（p=0.01 和 p<0.00001）。这些发现不支持任何特定的螯合治疗。文献显示存在偏倚风险，需要开展更多更大和更长时间的试验。
