Treatment of iron overload

Haema 2016; 7(3): 329-338

by Efthimia Vlachaki1, Alexandra Kourakli-Simeonidis2

1Assistant Professor of Hematology, Adult Thalassemia Unit, 2nd Department of Internal Medicine, Aristotle University, Hippokration Hospital, Thessaloniki, Greece,
2Hematologist, Director of Hematology Department of Department of Internal Medicine, Patra University Hospital, Greece

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Body Iron Overload results into hepatic, endocrinologic and cardiac complications which negatively affects quality of life and survival. Organ toxicity induced by iron overload could be prevent- ed or even partially reversed and both, overall survival and quality of life of iron overloaded patients can be improved through the use of effective iron chelation treatment. In the recent years, besides plasma ferritin levels, hepatic and cardiac iron load can be monitored by MRI, which helps to assess the efficacy of the iron chelation treatment. This assessment can drive the appropriate iron chelation strategy and dosage in the cases of severe transfusion-induced haemosiderosis. In hereditary haemochromatosis, phlebotomies constitute the cornerstone of treatment. In transfusion-induced (secondary) iron overload, which is the aim of this review, is usually observed in patients with β-Thalassaemia Major (β-τμ), sickle-cell anaemia, Myelodysplastic Syndromes, aplastic anaemia, or other rare causes of marrow failure, necessitating regular RBC transfusions. There are currently three available iron chelating agents. The development and the use of desferrioxamine, the first iron chelator, caused a revolution in the survival of the β-τμ patients. The use of desferrioxamine (DFO) has substantially prolonged overall survival in those patients. Unfortunately, the long term subcutaneous or intravenous infusion of DFO contributed to low compliance in those patients. Deferiprone (DFP), the first orally available iron chelator, turned out to be especially efficacious in the myocardial iron overload and furthermore, improved left ventricular ejection fraction (LVEF). This result is enhanced when DFP is co-administered with DFO. However, the need for three times daily regimen, along with the fear of neutropenia and arthralgias, makes it theoretically less appealing than the latest oral chelator, deferasirox. Deferasirox (DFX), due to its long half life, is administered once daily and has been proved as effective as DFO. It effectively lowers plasma ferritin and hepatic iron overload, while it also improves and protects myocardium in part by reducing plasma free toxic forms of iron down to normal levels. The most common adverse events with DFX therapy include gastrointestinal disturbances, rash, and mild increases in serum creatinine. However, compliance of chronic patients to their medication is problematic, thus its efficacy is impacted. Clinical trials are still ongoing, and they target to the development of an ideal iron chelation agent or even better of an agent, that will decrease the need for RBC transfusions in cases of ineffective erythropoiesis and hence will result to the limitation of iron overload.