A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

Inclusion Criteria: * Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity. * Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy. * Serum ferritin ≥ 8000 μg/L. * Serum ferritin \< 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight. * Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at \> 9 g/dL. * Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation. * Written, voluntary informed consent. Exclusion Criteria: * Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone). * Patients with non-transfusional hemosiderosis. * Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale. * Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening. * Patients with a history of nephrotic syndro…