Should Patients with Myelodysplastic Syndromes Undergo Iron Chelation Therapy? New Data Answering the Question

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Although it is well established that transfusional iron overload leads to organ impairment and shortened survival among children with thalassemia and sickle cell anemia, the role of transfusional iron overload and its treatment among older individuals with the myelodysplastic syndromes (MDS) remains unclear. Recent reviews have noted that MDS patients requiring frequent red blood cell transfusions experience higher rates of cardiac, hepatic, endocrine, and other organ damage in patterns similar to pediatric transfusional hemochromatosis, with increased rates of transformation to leukemia and decreased survival. Through the use of prognostic scoring systems such as the International Prognostic Scoring System (IPSS) and the World Health Organization (WHO) classification-based prognostic scoring system (WPSS), MDS patients with projected survivals long enough to warrant concern about the toxicities of iron overload can be identified and iron chelation therapies can be considered. The results of two large prospective trials among transfusion-dependent MDS patients, US03 and EPIC, have demonstrated that the oral iron chelation agent deferasirox can successfully reduce iron content and has an acceptable safety profile in this elderly population. Furthermore, retrospective trials have suggested that iron chelation therapy may prolong survival in MDS, and prospective trials are being planned. This article will highlight some of these issues.