Development and pathomechanisms of cardiomyopathy in very long-chain acyl-CoA dehydrogenase deficient (VLCAD(-/-)) mice

Biochim Biophys Acta. 2014 May;1842(5):677-85. doi: 10.1016/j.bbadis.2014.02.001. Epub 2014 Feb 12.

Abstract

Hypertrophic cardiomyopathy is a typical manifestation of very long-chain acyl-CoA dehydrogenase deficiency (VLCADD), the most common long-chain β-oxidation defects in humans; however in some patients cardiac function is fully compensated. Cardiomyopathy may also be reversed by supplementation of medium-chain triglycerides (MCT). We here characterize cardiac function of VLCAD-deficient (VLCAD(-/-)) mice over one year. Furthermore, we investigate the long-term effect of a continuous MCT diet on the cardiac phenotype. We assessed cardiac morphology and function in VLCAD(-/-) mice by in vivo MRI. Cardiac energetics were measured by (31)P-MRS and myocardial glucose uptake was quantified by positron-emission-tomography (PET). Metabolic adaptations were identified by the expression of genes regulating glucose and lipid metabolism using real-time-PCR. VLCAD(-/-) mice showed a progressive decrease in heart function over 12 months accompanied by a reduced phosphocreatine-to-ATP-ratio indicative of chronic energy deficiency. Long-term MCT supplementation aggravated the cardiac phenotype into dilated cardiomyopathy with features similar to diabetic heart disease. Cardiac energy production and function in mice with a β-oxidation defect cannot be maintained with age. Compensatory mechanisms are insufficient to preserve the cardiac energy state over time. However, energy deficiency by impaired β-oxidation and long-term MCT induce cardiomyopathy by different mechanisms. Cardiac MRI and MRS may be excellent tools to assess minor changes in cardiac function and energetics in patients with β-oxidation defects for preventive therapy.

Keywords: Dilated cardiomyopathy; Energy metabolism; MCT-supplementation; MRI; PET; VLCAD-deficiency.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acyl-CoA Dehydrogenase, Long-Chain / deficiency*
  • Acyl-CoA Dehydrogenase, Long-Chain / genetics
  • Animals
  • Cardiomyopathies / genetics
  • Cardiomyopathies / physiopathology*
  • Congenital Bone Marrow Failure Syndromes
  • Energy Metabolism
  • Gene Expression Regulation, Developmental
  • Gene Expression Regulation, Enzymologic
  • Glucose / metabolism
  • Lipid Metabolism, Inborn Errors / genetics
  • Lipid Metabolism, Inborn Errors / physiopathology*
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mitochondrial Diseases / genetics
  • Mitochondrial Diseases / physiopathology*
  • Muscular Diseases / genetics
  • Muscular Diseases / physiopathology*
  • Myocardium / metabolism
  • Polymerase Chain Reaction
  • Positron-Emission Tomography
  • Systole

Substances

  • Acyl-CoA Dehydrogenase, Long-Chain
  • Glucose

Supplementary concepts

  • VLCAD deficiency