| A pilot trial of deferiprone for neurodegeneration with brain iron accumulation. | |
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MedLine Citation:
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PMID: 21791473 Owner: NLM Status: Publisher |
Abstract/OtherAbstract:
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Deferiprone was shown to reverse iron deposition in Friedreich's ataxia. This multi-center, unblinded, single-arm pilot study evaluated safety and efficacy of deferiprone for reducing cerebral iron accumulation in neurodegeneration with brain iron accumulation. Four patients with genetically-confirmed pantothenate kinase-associated neurodegeneration, and two with parkinsonism and focal dystonia, but inconclusive genetic tests, received 15 mg/kg deferiprone bid. Magnetic resonance imaging and neurological examinations were conducted at baseline, 6 and 12 months. Chelation treatment caused no apparent hematologic or neurologic side effects. Magnetic resonance imaging revealed decreased iron accumulation in the globus pallidus of two patients (one with pantothenate kinase-associated neurodegeneration). Clinical rating scales and blinded video rating evaluations documented mild-to-moderate motor improvement in three patients (two with pantothenate kinase-associated neurodegeneration). These results underline the safety and tolerability of deferiprone, and suggest that chelating treatment might be effective in improving neurologic manifestations associated with iron accumulation. Clinicaltrials.gov identifier: NTC00907283. |
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Authors:
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Giovanni Abbruzzese; Giovanni Cossu; Manuela Balocco; Roberta Marchese; Daniela Murgia; Maurizio Melis; Renzo Galanello; Susanna Barella; Gildo Matta; Uberto Ruffinengo; Ubaldo Bonuccelli; Gian Luca Forni |
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Publication Detail:
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Type: JOURNAL ARTICLE Date: 2011-7-26 |
Journal Detail:
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Title: Haematologica Volume: - ISSN: 1592-8721 ISO Abbreviation: - Publication Date: 2011 Jul |
Date Detail:
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Created Date: 2011-7-27 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 0417435 Medline TA: Haematologica Country: - |
Other Details:
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Languages: ENG Pagination: - Citation Subset: - |
Affiliation:
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Italy; |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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