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The Cerebellum
Published in: The Cerebellum 1/2011

01-03-2011

Combined Therapy with Idebenone and Deferiprone in Patients with Friedreich’s Ataxia

Authors: Daniel Velasco-Sánchez, Asuncion Aracil, Raquel Montero, Ana Mas, Lorenzo Jiménez, Mar O’Callaghan, Maria Tondo, Antoni Capdevila, Josep Blanch, Rafael Artuch, Mercedes Pineda

Published in: The Cerebellum | Issue 1/2011

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Abstract

Iron chelators are a new therapeutical approach for patients with Friedreich’s ataxia, on the basis that oxidative cell damage that occurs in these patients is due to the increasing deposits of mitochondrial iron pools. The objective of the study was to evaluate the effects of the combined therapy of idebenone and low oral doses of deferiprone on the neurological signs and cardiac function parameters. This study was designed as a prospective open-label single-arm study. Twenty Friedreich’s ataxia patients were treated with idebenone (20 mg/kg/day) and deferiprone (20 mg/kg/day) for 11 months. Patients were evaluated before the start and throughout the study with the International Cooperative Ataxia Rating Scale (ICARS) scores, echocardiographic measurements and MRI (magnetic resonance imaging) techniques to asses brain iron deposits in the dentate nucleus. No significant differences were observed in total ICARS scores when comparing baseline status and the end of the study in the whole group of patients. Posture and gait scores increased significantly after 11 months of therapy (Wilcoxon’s test, p = 0.04) and kinetic function improved significantly (Wilcoxon’s test, p = 0.015). Echocardiography data showed a significant reduction of the interventricular septum thickness (Wilcoxon’s test, p = 0.04) and in the left ventricular mass index (Wilcoxon’s test, p = 0.038) after the start of the therapy. The MRI values in the dentate nucleus showed a statistically significant reduction (Wilcoxon’s test p = 0.007) between baseline conditions and after 11 months of the therapy. Combined therapy with idebenone and deferiprone in patients with FDRA indicates a stabilizing effect in neurologic dysfunctions due to an improvement in the kinetic functions, with a worsening of gait and posture scores. Heart hypertrophy parameters and iron deposits in dentate nucleus improved significantly. Combined therapy was well tolerated with mild side effects, apart from the risk of neutropenia and progressive reduction of plasma iron parameters.
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Metadata
Title
Combined Therapy with Idebenone and Deferiprone in Patients with Friedreich’s Ataxia
Authors
Daniel Velasco-Sánchez
Asuncion Aracil
Raquel Montero
Ana Mas
Lorenzo Jiménez
Mar O’Callaghan
Maria Tondo
Antoni Capdevila
Josep Blanch
Rafael Artuch
Mercedes Pineda
Publication date
01-03-2011
Publisher
Springer-Verlag
Published in
The Cerebellum / Issue 1/2011
Print ISSN: 1473-4222
Electronic ISSN: 1473-4230
DOI
https://doi.org/10.1007/s12311-010-0212-7

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