Deferiprone is the only orally active iron-chelating drug to be used therapeutically in conditions of transfusional iron overload.
Target：
Deferiprone is an orphan drug designed and developed primarily by academic initiatives for the treatment of iron overload in thalassaemia. Deferiprone has been used in several other iron or other metal imbalance conditions and has prospects of wider clinical applications. Deferiprone has high affinity for iron and interacts with almost all the iron pools at the molecular, cellular, tissue and organ levels. Doses of 50-120 mg/kg/day appear to be effective in bringing patients to negative iron balance. Deferiprone increases urinary iron excretion, which mainly depends on the iron load of patients and the dose of the drug.
Deferiprone is a potent iron chelating agent that is orally active, capable of penetrating the brain, cells, and skin. It is effective in inhibiting tumor cell proliferation and migration, while also promoting apoptosis. Additionally, deferiprone possesses anti-anemic, neuroprotective, anti-inflammatory, antioxidant, and antidotal properties, making it useful in studies related to cancer, cardiovascular disease, infection, inflammation, and neurological diseases[1].[2].[3].[4].[5].[6].[7].[8].
Deferiprone/去铁酮 动物研究
Animal study
Deferiprone (100 mg/kg daily for 4 weeks, ingested) demonstrates a neuroprotective effect in a mouse model of tauopathy, the rTg(tauP301L)4510[6].Deferiprine (50-200 mg/kg daily, orally for 5-10 days) alleviates nephrotoxicity in a rat model of acute renal failure induced by Cisplatin[7].Deferiprone (13.82 and 27.64 mg/kg per day, ingested for 4 weeks) exhibits anti-apoptotic and neuroprotective activities in a rat model of Alzheimer’s disease[8].
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