Deferasirox treatment in post-transfusion iron overload

Deferasirox has been approved in France since 2007 for the treatment of iron overload secondary to multiple transfusions in anaemic patients, principally in case of beta-thalassemia, sickle-cell anaemia or myelodysplastic syndrome. The present study describes the current state of knowledge and practice in deferasirox prescription and follow-up, with a view to improving efficacy, tolerance and medication adherence. Deferasirox is contra-indicated in case of renal insufficiency (creatinine clearance < 60 mL/min) or severe hepatic pathology. Dosage is initially determined by transfusion level and treatment objective (maintenance of iron balance or reduction of overload), then adapted according to efficacy and tolerance. Efficacy is assessed by measuring ferritinaemia and hepatic and cardiac overload, best quantified on MRI. In case of beta-thalassemia, regular MRI monitoring of cardiac overload is recommended after the age of 10-12 years. Side-effects are often dose-dependent and transitory, resolving without interruption of treatment. They mainly comprise gastrointestinal disorders, skin rash and mild to moderate blood creatinine level elevation. Patient education concerning the importance of treatment, the existence of side-effects and the means of preventing or limiting them should improve medication adherence.