Light and shadows in the iron chelation treatment of haematological diseases

Summary This review outlines the main chelator groups studied to date, and the evidence for their clinical effectiveness. For each treatment, the strength of evidence was documented according to the guidelines from the American College of Cardiology and the American Heart Association. Three main haematological diseases were considered as models: thalassaemia major, sickle‐cell disorders and myelodysplasia. Although the data in the literature do not allow firmly evidence‐based conclusions, the findings suggest that in thalassaemia major: (i) deferoxamine remains the drug of choice for chelation treatment; (ii) if there is deferoxamine intolerance or a change of treatment is suggested, the options are deferiprone or, if the liver iron concentration is high, deferasirox treatment; and (iii) if the ferritin level is >2500  μ g/l and liver iron concentation is >7 mg/g/dry weight, continuous subcutaneous (s.c.) or intravenous (i.v.) deferoxamine, or combined treatment with deferiprone and deferoxamine is advised. In case of heart failure, there is currently more solid documentation to support continuous s.c. or i.v. deferoxamine treatment than combined treatment with deferiprone and deferoxamine. However, more recent data in the literature suggest that the latter could be a satisfactory alternative. Finally, if iron chelation is required for sickle‐cell disorders or myelodysplastic syndromes, the current data support the use of deferoxamine treatment.