Low-dose idebenone treatment in Friedreich's ataxia with and without cardiac hypertrophy.
Left ventricular hypertrophy (LVH) is a frequent finding in Friedreich's ataxia (FRDA). In previous studies treatment with idebenone, a synthetic analogue of coenzyme Q10, has been associated with a substantial decrease in myocardial hypertrophy, despite great variability in cardiac responsiveness among patients. Here we present the results of a retrospective analysis of a cohort of 35 patients (20 with LVH, 15 without LVH) with confirmed molecular diagnosis of FRDA, treated with idebenone 5 mg/kg/day for up to five years. At the end of the study, we found an increase of interventricular septum and posterior wall thickness in the group without LVH before treatment and no change in the group with LVH before treatment. The neurological picture of the disease significantly deteriorated with time in both groups.
Department of Neurological Sciences, University of Federico II, 80131 Naples, Italy. rinaldi.ca@gmail.com
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Rinaldi
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Tucci
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Maione
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Giunta
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De Michele
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Filla
D000328:Adult; D000704:Analysis of Variance; D000975:Antioxidants / Q000627:therapeutic use*; D015331:Cohort Studies; D004452:Echocardiography; D005260:Female; D005500:Follow-Up Studies; D005621:Friedreich Ataxia / Q000150:complications / Q000188:drug therapy* / Q000473:pathology; D006321:Heart / Q000187:drug effects; D006346:Heart Septum / Q000000981:diagnostic imaging / Q000187:drug effects / Q000473:pathology; D006801:Humans; D017379:Hypertrophy, Left Ventricular / Q000150:complications / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D009206:Myocardium / Q000473:pathology; D012189:Retrospective Studies; D012720:Severity of Illness Index; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000627:therapeutic use
D000975:Antioxidants; D014451:Ubiquinone; C036619:idebenone