Peripheral sensory-motor polyneuropathy, pigmentary retinopathy, and fatal cardiomyopathy in long-chain 3-hydroxy-acyl-CoA dehydrogenase deficiency

Eur J Pediatr. 1992 Feb;151(2):121-6. doi: 10.1007/BF01958956.

Abstract

An 11-month-old girl presented acute episodes of hypoglycaemia and hepatic encephalopathy reminiscent of Reye syndrome and 3-hydroxydicarboxylic aciduria. The patient showed peculiar clinical manifestations of severe sensory-motor neuropathy, pigmentary retinopathy, and cardiomyopathy. She died of cardiac failure. Pathological studies of peripheral nerve showed signs of axonal neuropathy and demyelination. Enzymatic studies in cultured fibroblasts showed a deficiency of mitochondrial long-chain 3-hydroxyacyl-CoA-dehydrogenase. Peripheral nerve involvement and retinal pigmentary degeneration have as yet not been described in patients with proven defects of mitochondrial beta-oxidation.

Publication types

  • Case Reports

MeSH terms

  • 3-Hydroxyacyl CoA Dehydrogenases / deficiency*
  • Acyl-CoA Dehydrogenase, Long-Chain
  • Biochemical Phenomena
  • Biochemistry
  • Biopsy
  • Cardiomyopathy, Hypertrophic / complications*
  • Fatty Acid Desaturases / deficiency*
  • Female
  • Fibroblasts / enzymology
  • Heart Failure / etiology
  • Hereditary Sensory and Motor Neuropathy / complications*
  • Hereditary Sensory and Motor Neuropathy / diagnosis
  • Hereditary Sensory and Motor Neuropathy / pathology
  • Humans
  • Infant
  • Retinitis Pigmentosa / complications*
  • Sural Nerve / pathology

Substances

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