Iron Chelation Therapy

Deferiprone Treatment

• The FDA and EU approve the use of deferiprone, an oral iron chelator, to treat chronic iron overload in patients with β-thalassemia major, administered at 75-100 mg/kg/day divided into three daily doses 1, 2, 3, 4, 5
• Deferiprone functions as a bidentate ligand that binds iron in a 3:1 molar ratio to facilitate iron excretion primarily through urine (75-90%) 1, 2
• Critical monitoring requirement for deferiprone includes weekly neutrophil counts due to the risk of agranulocytosis, which occurs in approximately 1% of patients 1, 2, 3
• Common adverse effects of deferiprone include gastrointestinal symptoms, such as nausea, vomiting, and abdominal pain, as well as arthropathy and transient liver enzyme elevations 1, 2