Deferiprone as a Potential Treatment for Neurodegeneration with Brain Iron Accumulation

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Neurodegeneration with brain iron accumulation (NBIA) encompasses a genetically heterogeneous group of rare and progressive diseases with debilitating sequelae. The major clinical finding associated with these diseases is iron accumulation in the basal ganglia. This iron accumulation is thought to have a significant impact on disease symptomatology and progression. Deferiprone is an iron chelator that crosses the blood brain barrier and removes excess iron associated with the Parkinsonlike symptoms and the developmental delay. We conducted a literature search using Medline and EMBASE to identify efficacy and safety studies of deferiprone use in NBIA. Deferiprone showed the ability to significantly decrease brain iron with concomitant symptom improvement in some patients, but not in others. Because deferiprone has the potential to cause agranulocytosis and neutropenia, further studies are needed in order to determine its true safety and efficacy in NBIA. Standard methods for measuring brain iron and normal ranges for brain iron measurements are needed before the utility of brain iron chelation therapy can be quantified. Furthermore, the clinical benefits of deferiprone therapy and the patient population most likely to benefit from therapy needs to be determined by larger, randomized, studies.

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