Abstract
The long term consequences of iron toxicity are mostly reversible with effective iron chelation therapy. Recommendations for use of chelation therapy in transfusion dependent thalassaemia (TDT), sickle cell disease (SCD) and non transfusion dependent thalassaemia (NTDT) continue to evolve as our knowledge and clinical experience increases. Improved chelation options including drug combinations and a better understanding of condition specific factors may help to improve efficiency of chelation regimens and meet the needs of patients more effectively.
Highlights
The long term consequences of iron toxicity are mostly reversible ly without regular transfusions but many patients require intermittent with effective iron chelation therapy
The rate of iron loadchelation therapy in transfusion dependent thalassaemia (TDT), ing in SCD is influenced by the transfusion regimen, and sickle cell disease (SCD) and non transfusion dependent thalas- exchange transfusion regimens can significantly prevent risk of iron saemia (NTDT) continue to evolve as our knowledge and clinical experience increases
Improved chelation options including drug ly combinations and a better understanding of condition specific factors may help to improve efficiency of chelation regimens and meet n the needs of patients more effectively. se o Introduction l u There has been much progress in our understanding of iron overia load and its consequences and the aims of chelation therapy are focussed on the prevention of iron accumulation and irreversible c organ damage
Summary
Deferoxamine (DFO), Deferiprone (DFP), and Deferasirox (DFX) are currently licensed for treating iron overload in TDT, though with variations in age of initiation or as first/second line therapy depending on the licensing authority. Thalassemia Reports 2014; 4:4860 doi:10.4081/thal.2014.4860 junction with the patient or parents to continue or change to alternatives including combination therapies In this time it may be necessary to intensify treatments, e.g., in preparation for pregnancy when a peri-. Basic principles of regimens eration of combination therapy with DFO and DFP should be considered in patients with increasing myocardial iron. In TDT, most experience applies to use of DFO and DFP together but Initiation and continuation in children use of combinations of the oral iron chelators is gaining interest with favourable results reported in the literature. There is some evidence for the use of comfacilitated in practice by increasing high quality research exploring the bination therapy with DFO and DFP in children and this can be considefficacy, safety and tolerability of chelation regimens and monitoring ered as an option when first line therapy has failed.
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