Deferiprone

Deferiprone.svg

Deferiprone is an oral iron chelator used to treat iron overload in patients with thalassemia major and other conditions requiring frequent blood transfusions. It is particularly useful for patients who are unable to use deferoxamine due to its mode of administration or side effects.

Mechanism of Action

Deferiprone works by binding to excess iron in the body, forming a complex that is then excreted primarily through the urine. This helps to reduce the levels of iron in the body, preventing damage to vital organs such as the heart, liver, and endocrine glands.

Indications

Deferiprone is indicated for the treatment of transfusional iron overload in patients with thalassemia major when current chelation therapy is inadequate. It is also used in other conditions where iron overload is a concern, such as sickle cell disease and myelodysplastic syndromes.

Dosage and Administration

The dosage of deferiprone is typically based on the patient's body weight and the severity of iron overload. It is usually administered in divided doses, taken orally three times a day. Regular monitoring of iron levels and complete blood count is essential to adjust the dosage and ensure the effectiveness of the treatment.

Side Effects

Common side effects of deferiprone include nausea, vomiting, abdominal pain, and diarrhea. Serious side effects can include neutropenia and agranulocytosis, which require regular monitoring of the patient's white blood cell count. Other potential side effects include arthralgia, elevated liver enzymes, and zinc deficiency.

Contraindications

Deferiprone is contraindicated in patients with a known hypersensitivity to the drug or any of its components. It should also be used with caution in patients with a history of neutropenia or agranulocytosis.

Monitoring

Patients on deferiprone therapy require regular monitoring of their serum ferritin levels to assess the effectiveness of the treatment. Additionally, frequent monitoring of the absolute neutrophil count is necessary to detect early signs of neutropenia or agranulocytosis.

Pregnancy and Lactation

The use of deferiprone during pregnancy is not recommended unless the potential benefits outweigh the risks. It is unknown whether deferiprone is excreted in human milk, so caution should be exercised when administering the drug to breastfeeding mothers.

Research and Development

Ongoing research is exploring the use of deferiprone in other conditions associated with iron overload and its potential benefits in combination with other iron chelators.

See Also

• Thalassemia
• Iron overload
• Deferoxamine
• Sickle cell disease
• Myelodysplastic syndromes