A pilot trial of deferiprone for neurodegeneration with brain iron accumulation

被引：106

作者：

Abbruzzese, Giovanni ^{[3
]}

Cossu, Giovanni ^{[2
]}

Balocco, Manuela ^{[1
]}

Marchese, Roberta ^{[3
]}

Murgia, Daniela ^{[2
]}

Melis, Maurizio ^{[2
]}

Galanello, Renzo ^{[4
]}

Barella, Susanna ^{[4
]}

Matta, Gildo ^{[5
]}

Ruffinengo, Uberto ^{[6
]}

Bonuccelli, Ubaldo ^{[7
]}

Forni, Gian Luca ^{[1
]}

机构：

[1] Galliera Hosp, Ctr Microcitemia & Anemie Congenite Haematol, I-16128 Genoa, Italy

[2] G Brotzu Gen Hosp, Dept Neurol, Cagliari, Italy

[3] Univ Genoa, Dept Neurosci, I-16126 Genoa, Italy

[4] Univ Cagliari, Osped Reg Microcitemia, Cagliari, Italy

[5] G Brotzu Gen Hosp, Dept Radiol, Cagliari, Italy

[6] Galliera Hosp, Neuroradiol Unit, I-16128 Genoa, Italy

[7] Univ Pisa, Dept Neurosci, I-56100 Pisa, Italy

来源：

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL | 2011年 / 96卷 / 11期

关键词：

deferiprone; iron overload; neurodegeneration with brain iron accumulation;

D O I：

10.3324/haematol.2011.043018

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：1708 / 1711

页数：4

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