Exjade Benefits Chronically Transfused Patients

Ruzanna Harutyunyan's picture

New data from the largest prospective trial in iron chelation demonstrate the efficacy and safety of Exjade (deferasirox) in treating chronic transfusional iron overload, a potentially life-threatening condition for patients who have had multiple blood transfusions to treat underlying anemias, including beta-thalassemia and myelodysplastic syndromes (MDS).


-First prospective, multicenter study to show Exjade removes iron from the heart in beta-thalassemia patients with mild to severe cardiac iron overload

-In a subgroup analysis of 341 patients with myelodysplastic syndromes (MDS), Exjade significantly reduced levels of toxic iron

-These results are part of the largest prospective trial in iron chelation, which includes more than 1,700 patients with various transfusion-dependent anemias


Data from this landmark trial, known as EPIC, were presented today at the 50th American Society of Hematology (ASH) Annual Meeting and Exposition in San Francisco, California.

The EPIC cardiac substudy showed that Exjade removed iron from the heart in beta-thalassemia patients, based on a statistically significant improvement in T2* magnetic resonance imaging, a validated technique to assess cardiac iron content (P<0.0001). The one-year substudy included 114 beta-thalassemia patients with cardiac iron overload, the leading cause of death in these patients.

"These data clearly demonstrate that deferasirox significantly reduces cardiac iron in beta-thalassemia patients with iron overload, which is a critical goal of treatment for these patients," said Dudley Pennell, MD, Professor of Cardiology, Royal Brompton and Harefield NHS Trust and Imperial College, London. "Cardiac complications caused by the buildup of toxic iron in the heart can be life-threatening for people living with thalassemia."

A pre-planned analysis of 341 MDS patients enrolled in the study showed that Exjade significantly reduced levels of serum ferritin (SF), a key measure of iron in the body, by 253.0 ng/mL from baseline (P=0.0019). Of the 171 MDS patients whose SF was measured at one year, the decrease from baseline was 606 ng/mL.

"Many MDS patients receive regular blood transfusions as part of their ongoing treatment, which puts them at risk for iron overload," said Norbert Gattermann, MD, PhD, Hematology, Oncology and Clinical Immunology, Heinrich Heine University Medical Center, Dusseldorf, Germany. "This study, which includes the largest number of MDS patients of any iron chelation study, shows deferasirox can effectively reduce iron burden and is generally well tolerated when used appropriately to treat these patients."

Iron toxicity can lead to permanent damage of the liver, heart and endocrine glands, leading to an increased risk of serious health problems and early death. Previous studies of transfusion-dependent MDS patients have found that increased levels of SF are associated with shortened overall survival.