Medication Therapy for Treatment of Thalassemias

Thalassemia disease is a group of inherited autosomal recessive blood disorders, in which the body makes an abnormal form of hemoglobin, the protein in red blood cells that carries oxygen thus causing anemia, the characteristic presenting symptom of the thalassemias. Patient with thalassemia will get regular blood transfusion to treat his anemia, These transfusions can lead to a build-up of iron which can be harmful and this can cause serious side effects including diabetes, heart failure and liver disease.

The iron overload related to thalassemia may be treated by chelation therapy with medications such as Desferrioxamine, Deferiprone or Deferasirox. Medications therapy in thalassemia can be used to remove excess iron from the blood, This may have severe side effects and must be carefully monitored.

Deferoxamine is an intravenously administered chelation agent currently approved for use in the United States. This medicine only effective via daily injections which makes its long term use more difficult. It is also used with other medicines to treat acute iron poisoning, especially in small children. Deferoxamine (also known as desferrioxamine B, desferoxamine B, DFO-B, DFOA, DFB or desferal) is a bacterial siderophore produced by the actinobacteria Streptomyces pilosus. Using this medicine, It is common to develop pain, swelling, itchiness and redness at the site of the injection, nausea and headache.

Deferiprone is given as an oral medication. Generic name is (de-FER-i-prone), and brand Name is Ferriprox. The therapy is being approved under the U.S. Food and Drug Administration (FDA) in October 2011. Some side effects of deferiprone may not be reported, but some little cases of side effects include: Nausea, vomiting and diarrhea. Potentially, the most serious side effect of DFP is agranulocytosis (a condition where bone marrow no longer produces enough white blood cells).

Desferasirox has the benefit of being an oral medication. Brand name or marketed as Exjade. This medicine is indicated for the treatment of chronically elevated levels of iron in the blood caused by regular blood transfusions in thalassemias patient, especially for 2 years of age and older. It was approved by the United States Food and Drug Administration (FDA) in November 2005. By taking this medication, may risk to get side effects include nausea, vomiting, diarrhea, stomach pain, increases in kidney laboratory values, and skin rash.

The other medication therapy for thalassemia disease still under research. The long-term hope is that thalassemia major will be cured by insertion of the normal beta-chain gene through gene therapy or by another modality of molecular medicine. Source : Wikipedia, eMedicine, NHLBI, SienceDaily.