Pathology of skeletal muscle and impaired respiratory chain function in long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency with the G1528C mutation

Neuromuscul Disord. 1996 Oct;6(5):327-37. doi: 10.1016/0960-8966(96)00352-5.

Abstract

Lactic acidosis and mitochondrial abnormalities have been reported in long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency. We studied muscle morphology and the respiratory chain function in ten patients with LCHAD deficiency and the G1528C mutation. In eight cases the light microscopy of muscle specimens showed fatty infiltration and fibre degeneration. The degenerated fibres appeared as ragged red fibres in four cases. Electron microscopy revealed enlarged mitochondria often with swollen appearance in four out of seven patients. The number of mitochondria had also increased. Complex I associated enzyme activities in muscle mitochondria were decreased in five out of seven patients, and in three of them Complex II or II + III associated activities were also affected. We suggest that the reason for respiratory chain dysfunction and structural changes of mitochondria is the accumulation of toxic intermediates of fatty acid beta-oxidation in mitochondria. Because these changes may confound the differential diagnostics between LCHAD deficiency and respiratory chain defects, awareness of their frequency is important.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 3-Hydroxyacyl CoA Dehydrogenases / deficiency*
  • 3-Hydroxyacyl CoA Dehydrogenases / genetics*
  • Biopsy
  • Electron Transport / genetics
  • Fatty Acid Desaturases / deficiency
  • Humans
  • Infant
  • Infant, Newborn
  • Long-Chain-3-Hydroxyacyl-CoA Dehydrogenase
  • Microscopy, Electron
  • Mitochondria / enzymology
  • Mitochondria / ultrastructure
  • Muscle, Skeletal / enzymology*
  • Muscle, Skeletal / pathology*
  • Muscle, Skeletal / ultrastructure
  • Muscular Diseases / diagnosis*
  • Muscular Diseases / enzymology
  • Muscular Diseases / pathology
  • Point Mutation / physiology

Substances

  • 3-Hydroxyacyl CoA Dehydrogenases
  • Long-Chain-3-Hydroxyacyl-CoA Dehydrogenase
  • Fatty Acid Desaturases