Deferipron

Deferiprone, sometimes spelled deferipron in less common sources, is an oral iron chelating agent used to treat systemic iron overload in people who receive regular blood transfusions. It is particularly applied in conditions such as beta-thalassemia major and other anemias associated with transfusional iron loading, including situations where other chelators are not suitable or accessible. It is commonly used as part of a chelation strategy when deferoxamine is not feasible and may be used alone or in combination with other therapies depending on the clinical scenario and regional guidelines.

Mechanism of action

Deferiprone binds ferric iron (Fe3+) to form soluble complexes that are excreted from the body, primarily via the urine. By mobilizing iron from excess stores and circulating labile iron, the drug reduces total body iron burden and helps prevent iron-related organ damage. Because it acts on intracellular and extracellular iron pools, its effect can complement other chelators with different pharmacokinetic profiles.

Administration and dosing

Deferiprone is taken by mouth in divided doses, with a total daily dose typically around 75 mg/kg, administered in three portions. Dosing may be adjusted based on response, tolerability, and safety monitoring. It can be used for prolonged periods under close medical supervision, with adherence and timing important for effectiveness and safety.

Safety and monitoring

A major safety consideration is the risk of neutropenia and agranulocytosis, which requires regular blood monitoring, especially during initiation. Baseline and periodic complete blood counts are recommended, along with liver function tests and assessment of ferritin levels. Other possible adverse effects include gastrointestinal upset, joint and muscle pains, rash, and, less commonly, hepatic or renal issues. Use in pregnancy is generally avoided unless potential benefits justify risks.

Regulatory status and monitoring

Deferiprone is approved in many regions for transfusional iron overload in patients who cannot use other chelation therapies. Because of the neutropenia risk, patients on deferiprone typically undergo standardized monitoring and may be instructed to report signs of infection promptly.