A pilot trial of deferiprone for neurodegeneration with brain iron accumulation

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-co...

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Bibliographic Details
Published in:Haematologica (Roma) Vol. 96; no. 11; pp. 1708 - 1711
Main Authors: Abbruzzese, G., Cossu, G., Balocco, M., Marchese, R., Murgia, D., Melis, M., Galanello, R., Barella, S., Matta, G., Ruffinengo, U., Bonuccelli, U., Forni, G. L.
Format: Journal Article
Language:English
Published: Pavia Ferrata Storti Foundation 01.11.2011
Subjects:
Adult
Aged
Biological and medical sciences
Brief Reports
Female
Hematologic and hematopoietic diseases
Humans
Iron - metabolism
Iron Chelating Agents - administration & dosage
Iron Chelating Agents - adverse effects
Iron Metabolism Disorders - complications
Iron Metabolism Disorders - diagnostic imaging
Iron Metabolism Disorders - drug therapy
Iron Metabolism Disorders - physiopathology
Magnetic Resonance Imaging
Male
Medical sciences
Metabolic diseases
Metals (hemochromatosis...)
Middle Aged
Neurodegenerative Diseases - complications
Neurodegenerative Diseases - diagnostic imaging
Neurodegenerative Diseases - drug therapy
Neurodegenerative Diseases - metabolism
Other metabolic disorders
Pilot Projects
Pyridones - administration & dosage
Pyridones - adverse effects
Radiography
Iron overload
neurodegeneration with brain iron accumulation
Hematology
Central nervous system
Chelating agent
Metabolic diseases
Clinical trial
Deferiprone
Iron
Encephalon
ISSN:0390-6078, 1592-8721, 1592-8721
Online Access:Get full text
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Summary: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 2 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, six and 12 months. Chelation treatment caused no apparent hematologic or neurological side effects. Magnetic resonance imaging revealed decreased iron accumulation in the globus pallidus of 2 patients (one with pantothenate kinase-associated neurodegeneration). Clinical rating scales and blinded video rating evaluations documented mild-to-moderate motor improvement in 3 patients (2 with pantothenate kinase-associated neurodegeneration). These results underline the safety and tolerability of deferiprone, and suggest that chelating treatment might be effective in improving neurological manifestations associated with iron accumulation. (Clinicaltrials.gov Identifier: NTC00907283).
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-1
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ISSN:0390-6078
1592-8721
1592-8721
DOI:10.3324/haematol.2011.043018