Abstract
Many antioxidants have been suggested as potential treatments for Friedreich ataxia, but have not been tested in clinical trials. We found that a majority of patients in our cohort already use such antioxidants, including idebenone, which is not available at a pharmaceutical grade in the United States. Younger age, cardiomyopathy and shorter GAA repeat length were independent predictors of idebenone use, but no factors predicted use of other antioxidants. This confirms that non-prescription antioxidant use represents a major confounder to formal trials of existing and novel agents for Friedreich ataxia.
Publication types
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Clinical Trial
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Multicenter Study
MeSH terms
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Adolescent
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Adult
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Age Factors
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Age of Onset
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Antioxidants / administration & dosage*
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Cardiomyopathies / drug therapy
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Cardiomyopathies / genetics
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Cardiomyopathies / physiopathology
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Clinical Trials as Topic / standards
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Cohort Studies
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Confounding Factors, Epidemiologic
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Dose-Response Relationship, Drug
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Drug Therapy, Combination
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Female
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Friedreich Ataxia / drug therapy*
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Friedreich Ataxia / genetics
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Friedreich Ataxia / physiopathology
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Humans
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Male
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Middle Aged
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Mitochondria / drug effects
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Mitochondria / genetics
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Nonprescription Drugs / therapeutic use*
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Oxidative Stress / drug effects
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Oxidative Stress / genetics
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Patient Selection
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Placebo Effect
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Self Medication / statistics & numerical data*
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Self Medication / trends
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Trinucleotide Repeat Expansion / drug effects
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Trinucleotide Repeat Expansion / genetics
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Ubiquinone / administration & dosage
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Ubiquinone / analogs & derivatives*
Substances
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Antioxidants
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Nonprescription Drugs
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Ubiquinone
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idebenone