Iron Chelation & b-Thalassemia
Beta thalassemias is an inherited blood disorder that results from the reduced or absent synthesis of the beta chains of hemoglobin. The ineffective and low levels of hemoglobin usually result in the development of anemia. This anemia will put the patients under stress to keep their hemoglobin level at a normal level through regular and lifelong blood transfusions, otherwise, patients will suffer from severe anemic symptoms and death in certain cases.
While the treatment of thalassemia is straightforward through regular blood transfusion, another major issue emerges as a result of blood transfusion which is iron overload. Unfortunately, the human body can regulate iron only through controlling iron absorption from food but can’t excrete an excessive amount of iron. That’s why beta-thalassemia patients should be on iron chelation to control their iron level in blood, liver, endocrine system, and heart.
Usually, iron chelators are prescribed to beta-thalassemia patients when their serum ferritin reaches 1000 ng/mL or when they get 10 to 20 blood transfusions. The target is to keep these patients with a serum ferritin below 500 ng/mL and to avoid over chelation as it may affect both vision and Auditory scene.
Recent studies proposed that iron overload starts in blood as the serum ferritin starts to elevate, followed by accumulation of iron in the liver and finally heart and endocrine system. Moreover, around 10% of beta-thalassemia patients die from heart failure due to improper iron chelation or low adherence to chelation therapy by patients.
Currently, there are three (3) approved chelators for beta-thalassemia:
1- Desferal parenteral route
2- Deferiprone (Oral therapy)
3- Deferasirox (Oral therapy)
