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Once-Daily Exjade Removes Toxic Iron From Heart, Liver

Armen Hareyan's picture

New data show once-daily Exjade (deferasirox) reduces life-threatening iron levels in the heart and liver in beta-thalassaemia patients. These interim results from an ongoing trial show that at, six months, approximately 78 percent of participants had decreases in cardiac iron and 90 percent of patients had decreases in hepatic iron. These results were reported at the 49th Annual Meeting of the American Society of Haematology (ASH) in Atlanta, 8-11 December 2007.

Chronic iron overload is a potentially life-threatening condition that results from frequent blood transfusions required to treat certain types of chronic blood disorders, including sickle cell disease (SCD), thalassaemia, and myelodysplastic syndromes (MDS) and other anaemias. If left undiagnosed or untreated, excess iron in the body can become toxic, causing damage to the heart and liver, ultimately leading to death.

"These preliminary data are very encouraging. At a dose of 30 mg/kg, Exjade lowered both heart and liver iron levels in most patients," said Dr Farrukh Shah, Consultant Haematologist, The Whittington Hospital NHS Trust. "These data add to the growing body of evidence that Exjade successfully removes iron from the heart. This is particularly important because iron cardiotoxicity remains the leading cause of death in thalassaemia major patients."

The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion-related iron overload.(5) Prior to oral iron chelation, patients relied on chelation therapy that had to be administered by nightly infusions by needle and pump, often lasting 8-12 hours a night for 5 to 7 nights a week. Once-daily Exjade oral monotherapy offers patients effective iron reduction, without the need for any infused chelation therapy.

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Long-term efficacy and safety demonstrated in sickle cell disease Patients

Interim results after 2.7 years of a separate four-year extension study demonstrates the long-term, dose-dependent efficacy and safety of treatment with Exjade for chronically transfused patients with SCD. Patients treated in the core study with doses of 20 and 30 mg/kg/day showed continued decline in serum ferritin (SF), an indication of iron buildup in the body. For patients initially treated with 5 and 10 mg/kg/day doses in the core study, SF levels gradually declined following a dose increase to approximately 20 mg/kg/day. There were no significant changes in markers of liver or renal function and no cases of progressive increases in serum creatinine (SCr). Additionally, no new adverse events or safety concerns have been reported so far in the extension study.

Exjade demonstrates safety and efficacy in lower-risk MDS patients

Additional research demonstrated that treatment with Exjade decreased mean SF levels over one year in patients with low- or intermidiate-1 IPSS risk MDS. Additionally, 100 percent of patients experienced a stabilised labile plasma iron (LPI) over 12 months, indicating 24-hour sustained suppression of toxic iron with Exjade. Exjade was shown to have a manageable safety profile in this population. Ongoing assessments of this trial, evaluating cardiac, hepatic and endocrine function, will determine the impact of iron reduction with Exjade on morbidity and mortality in MDS.

These data provide further context to a separate study presented at this year's meeting, which demonstrates that chelation therapy provides a significant survival benefit for heavily-transfused patients with low and intermediate MDS. A prospective study of MDS patients found that the median overall survival from diagnosis was 115 months in chelated patients versus 51 months in non-chelated patients (p less than 0.0001).