New Clinical Data Shows Innovative Treatment Removes Iron from Heart
Regional doctors, healthcare administrators, patients, and parents attending the 12th International Thalassemia International Federation (TIF) Conference in Dubai today heard that the new oral therapy to remove excess iron from the bodies of thalassemia sufferers has been shown as effective and safe from the accumulating clinical data.
Also, in several preclinical and clinical studies, the treatment, Exjade was shown to effectively remove excess iron accumulated in the heart. Accumulated cardiac iron is known to be a leading cause of heart failure and death in frequently transfused thalassemia patients.
Additional data from a separate study in sickle cell disease patients also confirms the efficacy of Exjade in removing excess total body and liver iron.
“The important findings of these studies were shared today at TIF, and the news that Exjade is a safe, effective, and more convenient alternative to chelation therapy was very well received by doctors, but also very encouraging for patients and parents,” said Dr. Abdullah Al Khayat, Director of Dubai’s Al Wasl Hospital and Thalassemia Center.
“The Middle East sits squarely within the thalassemia belt, with some GCC countries experiencing thalassemia gene carrier rates of as high as one in ten residents. With Exjade, attending physicians now have a strong weapon with which to combat the complications of this menacing disease, before it can cause severe and lasting organ damage,” added Dr. Al Khayat.
About Iron Overload and Iron Chelation
Iron overload is a cumulative, potentially life-threatening, unavoidable consequence of chronic blood transfusions used to treat certain types of rare chronic blood disorders, including thalassemia and sickle cell disease, as well as other rare anemia and myelodysplastic syndromes. Signs of iron overload may be detected after transfusion of about 20 units of blood. In many patients the need for transfusions may be life-long. If left undiagnosed or untreated, the excess iron in the body is likely to lead to damage to the liver, heart and endocrine glands.
The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion related iron overload. In iron chelation, an agent binds to iron in the body and tissues and helps remove it through the urine and/or faeces.
Photo caption: Dr. Walid Kattouha, region head, EEMEA, Novartis Oncology, who spoke at the event.
Approved in November 2005 in the United States and Switzerland, Exjade is the first and only once-daily oral iron chelator and is administered as a drink. To date, deferoxamine is the standard of care for the first-line treatment of transfusional iron overload in most countries around the world. Administration of deferoxamine often requires a subcutaneous infusion, lasting eight to 12 hours per night for five to seven nights a week for as long as the patient continues to receive blood transfusions or has excess iron within the body. Depending on the disease, patients may require hundreds of these infusions each year. As a result, many patients may have stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload.
Mild, non-progressive increases in serum creatinine, mostly within the normal range, occur in about one-third of Exjade treated patients. These are dose-dependent, often resolve spontaneously and can sometimes be alleviated by reducing the dose. Serum creatinine should be assessed before initiating therapy and should be monitored monthly thereafter to determine if dose modification or discontinuation is necessary. Liver function should be monitored monthly and if there is an unexplained, persistent, or progressive increase in serum transaminase levels Exjade should be interrupted or discontinued.
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