Liver deferasirox

The oral iron chelator deferasirox is approved for treatment of iron overload. Deferasirox appears to be as effective as deferoxamine at reducing liver iron concentrations and is much more convenient. However, it is not clear yet whether deferasirox is as effective as deferoxamine at protecting the heart from iron overload. 

While the bulk of literature on iron chelation concerns P-thalassemia, in patients with thalassemia intermedia abnormal regulation of iron homeostasis may lead to iron overload, even in the absence of transfusions. In an open study in 11 patients with thalassemia intermedia, deferasirox 10-20 mg/kg/day for 24 months was associated with significant reductions in liver iron content and serum ferritin concentrations in the first 12 months, which continued during the second part of the study no serious adverse events were recorded [9 ]. 

In another patient thrombocytopenia due to deferasirox developed as part of a generalized delayed hypersensitivity reaction, in combination with a rash, fever, eosinophilia, renal impairment, and liver injury [13" ]. 

In a cost-utility multiple appraisal of deferasirox, deferoxamine, and deferiprone, it was concluded that in the short term there is little clinical difference between any of the three chelators in terms of removing iron from the blood and the liver, and that deferasirox may be cost-effective compared with deferoxamine but not compared with deferiprone [15 ]. The authors emphasized that the primary focus for future research should be on the longterm benefits of chelation therapy, including adverse reactions and adherence. 

Liver The data sheet for Exjade (deferasirox, Novartis) has been revised and a boxed warning has been added that hepatic failure can occur and that deaths have occurred [17, 18 ]. 

In one study, deferasirox treatment reduced total body iron but not liver iron [33 ]. Thirty patients receiving 25-35 mg/kg/day for 18 months had significant decreases in serum ferritin, but did not experience significant... 

A Cochrane Database of Systematic Reviews meta-analysis of 22 trials including 2187 participants of deferoxamine mesylate for the treatment of iron overload in people with transfusion-dependent thalassaemias was performed [47 ]. Adverse events were recorded in 18 trials. Five trials reported a total of seven deaths, three in patients with defroxamine alone and two in patients who received deferoxamine and deferiprone. Seven trials reported cardiac function or liver fibrosis in end organ damage. The authors concluded that there is a need for adequately powered, high-quality trials comparing the overall clinical efficacy and long-term outcomes of deferiprone, deferasirox and deferoxamine. 

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