an oral drug has been approved for treating Thalassemia patients with excess
iron due to blood transfusions. Thalassemia, a genetic blood disorder that
causes anemia, necessitates the affected patient to have frequent blood
transfusions. This leads to a serious, often fatal condition called iron
overload. Excess iron can have a number of deleterious effects on the body. The risk of
developing liver disease, diabetes, arthritis, heart failure, or an abnormal
heart rhythm is higher in patients with iron overload.
iron overload is usually treated with chelation therapy. Chelation therapy uses
chemical agents that can remove heavy metals from the body. The modality does not work for all patients. Ferriprox has
been approved for use when chelation therapy is inadequate.
from 12 clinical studies in 236 patients were analysed. Patients participating
in the study did not respond to previous iron chelation therapy. Deferiprone
was found to bring a 20% decrease in serum ferritin, a protein that stores iron
in the body for later use. The
drug was found to successfully reduce ferritin levels in half of the patients in the study.
U.S. Food and Drug Administration (FDA), has listed the following side effects
Abdominal and joint pain
Urine discoloration (chromaturia)
A decrease in the number of white blood cells (neutropenia)
An increase in the level of a liver enzyme that may be
indicative of tissue or liver damage at unsafe amounts
patients treated with Ferriprox developed a serious and potentially
life-threatening condition called agranulocytosis. The number of granulocytes
(a type of white blood cell that fights infection) falls dangerously in
uses of the drug are being probed into. ApoPharma, the company behind
Ferriprox, has committed to study the effect of the drug in patients with
sickle cell disease who have transfusional iron overload.
Food and Drug Administration (FDA)