Deferiprone: A Metabolic Manipulator for Iron Overload

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Metabolic Manipulator
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1 Identification , structure and class of manipulator
The name of the metabolic manipulator is deferiprone which belongs to the class of 4- pyridines.
It is substituted by methyl group at position 1 and 2 and by hydroxyl group at position 3.
Deferiprone has the following effects on organisms; nausea, vomiting, stomach, back pain.
It also causes a decrease in the number of white blood cells a condition known as neutropenia
and an increase in the level of enzymes found in the liver such as alanine transaminase and
alkaline phosphatase, an indicative of liver damage or tissue damage. Deferiprone is a chelating
agent that has a high affinity for ferric ion and thus binds to ferric ions in the body to form a
stable complex substance. Its mechanism of action is referred to as iron chelating activity and by
binding to iron, it is able to remove excess iron in the body. Deferiprone binds to plasma protein
and the enzyme involved in activity chelating activity is UGT1A. The enzyme metabolizes
deferiprone to 3-O-glucuronide. The half-life of deferiprone is 1.9 hours. The process can cause
agranulocytosis and neutropenia which can lead to hepatoxicity and fatal infections.

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2. Research
Several studies have been carried out to identify therapeutic potentials of deferiprone, its
biochemical pathways and characterization of its metabolic status. One of the most recent studies
on deferiprone was carried out in the United States to find out whether deferiprone is effective in
the treatment of neurodegeneration with brain iron accumulation disorder (NBIA) (4,3). The
disorder is characterized by an accumulation of iron in the brain and the progressive
degeneration of the nervous system, a condition known as basal ganglia. As per the study, the
disorder is very common to children. According to the research, iron chelator therapy which has
the ability to effectively cross blood-brain barrier has been proposed as one of the possible
therapies for NBIA.
Deferiprone is effective oral iron chelator which is utilized internationally in the curing of
iron overload. In regards to clinical studies, deferiprone effectively crosses the blood-brain
barrier and has a radiographic and clinical efficacy in symptomatic patients with excess iron in
brain cells. According to the results of this research, deferiprone was found to be effective in the
management of this disorder.
The other research regarding deferiprone is a comparative study of deferiprone and
silymarin versus deferiprone and placebo. The objectives of the study were to compare the iron
chelating efficacy of combination therapy of oral deferiprone. The research was carried out on
forty children who had beta thalassemia, in Tanta university hospital. The study took place from
October 2012 to October 2013. Patients who were suffering from thalassemic were grouped into
two by simple random allocation. The first group received a combination of daily oral