Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial.
Neurology
; 82(7): 607-13, 2014 Feb 18.
Article
en En
| MEDLINE
| ID: mdl-24453079
ABSTRACT
OBJECTIVE:
To assess whether bezafibrate increases fatty acid oxidation (FAO) and lowers heart rate (HR) during exercise in patients with carnitine palmitoyltransferase (CPT) II and very long-chain acyl-CoA dehydrogenase (VLCAD) deficiencies.METHODS:
This was a 3-month, randomized, double-blind, crossover study of bezafibrate in patients with CPT II (n = 5) and VLCAD (n = 5) deficiencies. Primary outcome measures were changes in FAO, measured with stable-isotope methodology and indirect calorimetry, and changes in HR during exercise.RESULTS:
Bezafibrate lowered low-density lipoprotein, triglyceride, and free fatty acid concentrations; however, there were no changes in palmitate oxidation, FAO, or HR during exercise.CONCLUSION:
Bezafibrate does not improve clinical symptoms or FAO during exercise in patients with CPT II and VLCAD deficiencies. These findings indicate that previous in vitro studies suggesting a therapeutic potential for fibrates in disorders of FAO do not translate into clinically meaningful effects in vivo. CLASSIFICATION OF EVIDENCE This study provides Class I evidence that bezafibrate 200 mg 3 times daily is ineffective in improving changes in FAO and HR during exercise in adults with CPT II and VLCAD deficiencies.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Bezafibrato
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Carnitina O-Palmitoiltransferasa
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Músculo Esquelético
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Enfermedades Mitocondriales
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Acil-CoA Deshidrogenasa de Cadena Larga
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Ácidos Grasos
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Errores Innatos del Metabolismo Lipídico
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Enfermedades Musculares
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Hipolipemiantes
Tipo de estudio:
Clinical_trials
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Diagnostic_studies
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Guideline
Límite:
Adolescent
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Adult
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Aged
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
Neurology
Año:
2014
Tipo del documento:
Article
País de afiliación:
Francia