ICL670: clinical outcome

In thalassemia, myelodysplastic syndrome (MDS) and sickle cell disease (SCD), long-term substitution therapy for anemia results in toxic iron overload which constitutes a significant medical problem. As humans have no physiological mechanism to excrete excess iron, this is deposited in the form of ferritin and hemosiderin in the liver, spleen, many endocrine organs and in the myocardium. This accumulation results in a host of clinical complications such as heart disease, diabetes, hypothyroidism and liver failure, and the primary causes of death among patients who require regular transfusions are due to the effects of iron overload rather than the underlying problem.