Comparative Efficacy and Safety of Deferoxamine, Deferiprone and Deferasirox on Severe Thalassemia: A Meta-Analysis of 16 Randomized Controlled Trials

Abstract

ObjectiveA 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. MethodsRandomized 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).ResultsSixteen 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).ConclusionFindings indicated that the most effective and safe iron chelators remains to be proven, and further large-scale, long-term studies are needed.

Effect of intravenous Deferoxamine concomitant use with blood transfusion on serum ferritin in thalassemia major patients

“…[17][18][19] In our study after intervention the usage of subcutaneous Deferoxamine in average was significantly decreased followed by reduction in the amount of medication side effects and health care costs. 20 Mashhadi et al showed that the concomitant use of Deferoxamine and Deferiprone was effective in reducing ferritin values but the symptoms such as dysplasia, nausea, joint pain and stiffness were seen in 13-18 % of the patients. 19 It is noted that none of the patients in our study suffered from acute complication of intravenous Deferoxamine and were not excluded due to complications caused by Deferoxamine in follow up period.…”

CONIFER - Non-Interventional Study to Evaluate Therapy Monitoring During Deferasirox Treatment of Iron Toxicity in Myelodysplastic Syndrome Patients with Transfusional Iron Overload

“…However, given the exploratory nature of the study, the heterogeneity of the study cohort and the high rate of concomitant disease, these results must be interpreted with caution. Furthermore, several controlled trials as well as meta-analysis confirmed the safety and effectiveness of deferasirox and the current study showed no new safety signals [9,13,25,26]. Nevertheless, a monthly monitoring of renal parameters is recommended for patients with normal renal function, whereas weekly monitoring is recommended in patients with pre-existing renal impairment [23].…”

Effectiveness and Safety of Deferasirox in Thalassemia with Iron Overload: A Meta-Analysis

“…Another systemic review by Xia et al [9] evaluated the effects of DFO, deferiprone, and DFX in severe thalassemia cases and found an increase in SF levels in the 5 and 10 mg/kg groups of DFX, but they were unchanged for those who received 20 mg/kg DFX and were reduced after administration of 30 mg/kg. They suggested that effectiveness of DFX in reducing SF levels may require appropriate dose adjustment.…”

“…Whether DFX can replace DFO to become the first-line treatment drug remains debatable. According to previous meta-analyses [8, 9], the few existing studies have reported inconclusive data. Since then, a few large-scale studies have recently been completed.…”