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.