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.
| Original language | English |
|---|---|
| Journal | Neurology |
| Volume | 82 |
| Issue number | 7 |
| Pages (from-to) | 607-13 |
| Number of pages | 7 |
| ISSN | 0028-3878 |
| DOIs | |
| Publication status | Published - 18 Feb 2014 |
Keywords
- 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