Document Detail

Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial.
MedLine Citation:
PMID:  24453079     Owner:  NLM     Status:  MEDLINE    
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.
Mette Cathrine Ørngreen; Karen Lindhardt Madsen; Nicolai Preisler; Grete Andersen; John Vissing; Pascal Laforêt
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Publication Detail:
Type:  Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't     Date:  2014-01-22
Journal Detail:
Title:  Neurology     Volume:  82     ISSN:  1526-632X     ISO Abbreviation:  Neurology     Publication Date:  2014 Feb 
Date Detail:
Created Date:  2014-02-18     Completed Date:  2014-04-18     Revised Date:  2014-08-19    
Medline Journal Info:
Nlm Unique ID:  0401060     Medline TA:  Neurology     Country:  United States    
Other Details:
Languages:  eng     Pagination:  607-13     Citation Subset:  AIM; IM    
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MeSH Terms
Acyl-CoA Dehydrogenase, Long-Chain / blood,  deficiency*,  metabolism
Bezafibrate / administration & dosage,  blood,  pharmacology*
Carnitine O-Palmitoyltransferase / deficiency*
Clinical Protocols
Cross-Over Studies
Fatty Acids / blood,  metabolism*
Hypolipidemic Agents / administration & dosage,  blood,  pharmacology*
Lipid Metabolism, Inborn Errors / blood,  diagnosis,  drug therapy*,  metabolism*
Middle Aged
Mitochondrial Diseases / blood,  diagnosis,  drug therapy*,  metabolism*
Muscle, Skeletal / drug effects,  metabolism*
Muscular Diseases / blood,  diagnosis,  drug therapy*,  metabolism*
Treatment Outcome
Young Adult
Reg. No./Substance:
0/Fatty Acids; 0/Hypolipidemic Agents; EC Dehydrogenase, Long-Chain; EC O-Palmitoyltransferase; Y9449Q51XH/Bezafibrate

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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