New study results support access to breakthrough treatment for transfusion-dependent anemia patients

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TORONTO, Dec. 9 /CNW/ – New study results presented at the American Society of Hematology (ASH) meeting confirming the efficacy of Exjade(R) in treating iron overload are welcomed by patients with this potentially life-threatening condition. This study is the largest international trial ever conducted with an iron chelator (a treatment that removes excess iron from the blood). The results show Exjade is safe and efficacious for patients with all types of chronic anemia, including thalassemia, sickle cell disease, and other rare forms of anemia. As importantly, this is the first study to demonstrate
that for beta-thalassemia patients with mild to severe iron overload in the heart, Exjade removes excess cardiac iron, the leading cause of death in this patient population.

“The strength of these results should eliminate the last barrier to
Exjade in Canada,” said Durhane Wong-Rieger, president and CEO of the Anemia Institute for Research and Education. “While transfusion-dependent anemia patients in most other countries have had access to an oral iron chelator for years, in Canada, provincial drug plans have asked for larger scale studies over a longer period of time. With these study results, there is no longer any reason for provincial governments to limit access to Exjade.”

Frequent blood transfusions are essential for patients to manage various blood disorders, including thalassemia, sickle cell disease and other rare forms of anemia. Every transfused unit of red blood cells contains roughly a six-month supply of iron, which the body cannot eliminate on its own. Patients receiving frequent blood transfusions, if untreated, can develop iron overload, which may damage major organs and eventually shorten life. To treat iron overload, patients must take a drug that removes iron from the bloodstream – called iron chelation therapy. Exjade is the only oral iron chelation therapy approved by Health Canada. Prior to Exjade, the only iron chelation therapy available in Canada was a drug that is infused very slowly into the body by a needle attached to a pump over a period of eight to 12
hours per day.

“In Canada and the US, Exjade is the first treatment breakthrough in iron chelation therapy in 40 years, and the only oral iron chelator available, bringing much-needed choice to patients and physicians,” said Dr. Isaac Odame, a hematology and oncology specialist at oronto’s Hospital for Sick Children who treats all forms of rare anemias. “These results, from the largest prospective trial of any iron chelator, further confirm the efficacy and safety of Exjade. This is particularly exciting for patients with the rarest forms of anemia, as these are some of the first results we’ve seen.”
Nesrin Berrak, a thalassemia patient from Toronto knows first-hand the
importance of ongoing research into treatments that help patients with
transfusion-dependent diseases stay healthy.

“Every time I go to the clinic for treatment, I see patients like me who
are fighting the same issues of transfusion and iron overload, but who suffer from other rare forms of anemia,” said Ms. Berrak. “I am very pleased that this new study supports the use of Exjade to treat iron overload in these conditions, as well as in thalassemia and sickle cell disease. In Ontario, these results should convince the government to make Exjade available to all patients who need it, without discrimination based on form of anemia.”

About the Anemia Institute for Research and Education

The Anemia Institute is a non-profit organization dedicated to generating and sharing knowledge about anemia as a serious condition – particularly amongst patients and health care professionals dealing with disease and/or treatment related risks factors for anemia. For more information on anemia and other blood disorders, please visit

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