Regular blood transfusions provide thalassemia patients with the red blood cells they need to survive. Once these red blood cells are broken down, however, the body is left with an excess of iron.
Although iron is essential to the body, excess iron can lead to organ failure and death. It is necessary that this excess iron be removed, or chelated, because it stores in the vital organs of the body, such as the heart and liver.
Nowadays, drugs designed to remove excess iron (iron chelators) have significantly changed the prognosis of thalassemia major. Patients can grow and develop normally, with relatively normal heart and liver functions. Adult patients are living into their forties and some have children of their own.
In Canada, there are currently two drugs approved to treat iron overload – Desferal and Exjade.
Desferal (deferoxamine) is an effective medication for removing iron from the body. It must be administered slowly by needle, in the vein or under the skin, for eight to 12 hours per day, five to seven days a week. Desferal has significantly changed the prognosis of patients with thalassemia major, but many of the patients find the infusions of Desferal difficult or painful and are reluctant to comply with their doctor’s orders. These patients are not free of risk and may die prematurely due to iron overload.
Exjade (deferasirox) is the first oral iron chelator available in Canada. It is an effective, well-tolerated pill that a patient dissolves in water and drinks once a day. Exjade improves patients’ quality of life and may lead to improved patient compliance with treatment.
The Guidelines for the Clinical Care of Patients with Thalassemia in Canada are now available. The Guidelines offer patients and parents of patients with thalassemia with a concrete document that they can refer to, to both ensure–and demand–they receive the best possible care, management, and treatment available. To download a copy of the guidelines, please click here.
Please talk with your doctor about which treatment is right for you.
For more information on financial support for thalassemia patients, please click here.