Bezafibrate in skeletal muscle fatty acid oxidation disorders: A randomized clinical trial
Research output: Contribution to journal › Journal article › Research › peer-review
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.
Original language | English |
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Journal | Neurology |
Volume | 82 |
Issue number | 7 |
Pages (from-to) | 607-613 |
Number of pages | 7 |
ISSN | 0028-3878 |
DOIs | |
Publication status | Published - 18 Feb 2014 |
- Acyl-CoA Dehydrogenase, Long-Chain, Adolescent, Adult, Aged, Bezafibrate, Carnitine O-Palmitoyltransferase, Clinical Protocols, Cross-Over Studies, Fatty Acids, Female, Humans, Hypolipidemic Agents, Lipid Metabolism, Inborn Errors, Male, Middle Aged, Mitochondrial Diseases, Muscle, Skeletal, Muscular Diseases, Treatment Outcome, Young Adult
Research areas
ID: 138139662