Abstract

The majority of transfusion-dependent patients with myelodysplastic syndromes (MDS) develop iron overload, which can lead to the accumulation of labile plasma iron (LPI) and cellular labile iron pool (LIP). These iron species catalyze the generation of reactive oxygen species (ROS), which are responsible for oxidative cellular damage and progressive organ dysfunction. This single-arm, open-label trial investigated the effects of deferasirox (Exjade®), a once-daily oral iron chelator, on parameters of oxidative stress and iron overload in iron-overloaded patients with low-risk MDS. Nineteen patients completed a mean 91 days' deferasirox treatment. There were significant reductions in mean ROS and lipid peroxidation in red blood cells (RBC), and significant increases in reduced glutathione in RBC and platelets. Moreover, mean LIP in RBC and platelets, and LPI, were decreased significantly. This study demonstrates the potential of deferasirox to act as an antioxidant by decreasing intra- and extra-cellular toxic iron species and oxidative stress parameters.

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