[Atypical course of a multiple acyl-CoA-dehydrogenase deficiency]

Klin Padiatr. 1999 Sep-Oct;211(5):413-6. doi: 10.1055/s-2008-1043823.
[Article in German]

Abstract

In a female newborn presenting with rapid metabolic deterioration (hypoketotic hypoglycaemia and acidosis) clinically accompanied by a "sweaty feet"-odour, the excretion pattern of organic acids in the urine suggested on the fourth day of live multiple acyl-CoA-dehydrogenase-deficiency, a potentially lethal autosomal-recessively inherited inborn error of fatty acid beta-oxidation and of the metabolism of certain amino acids. Diagnosis was confirmed by tandem-mass-spectrometry of acyl-carnitines in blood. Despite the poor prognosis of neonatal-onset multiple acyl-CoA-dehydrogenase-deficiency, treatment with carnitine, riboflavine, and a high-energy diet low in fat and high in carbonhydrates resulted in clinical stabilization. The infant survived various infection-associated decompensations and developed satisfyingly up to the age of 15 months, when another metabolic crisis resulted in multiorgan failure and death.

Discussion: Patients with neonatal-presenting multiple acyl-CoA-dehydrogenase-deficiency but without severe malformations may survive the first months of life. Tandem mass-spectrometry is a suitable tool to differentiate between multiple acyl-CoA-dehydrogenase-deficiency and other defects of fatty acid beta-oxidation.

Publication types

  • Case Reports
  • English Abstract
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylcarnitine / blood
  • Acyl-CoA Dehydrogenase
  • Acyl-CoA Dehydrogenases / deficiency*
  • Acyl-CoA Dehydrogenases / genetics
  • Amino Acid Metabolism, Inborn Errors / diagnosis*
  • Amino Acid Metabolism, Inborn Errors / enzymology
  • Amino Acid Metabolism, Inborn Errors / genetics
  • Amino Acid Metabolism, Inborn Errors / therapy*
  • Carnitine / administration & dosage
  • Diagnosis, Differential
  • Dietary Carbohydrates / administration & dosage
  • Dietary Fats / administration & dosage
  • Energy Intake
  • Fatal Outcome
  • Female
  • Humans
  • Infant, Newborn
  • Lipid Metabolism, Inborn Errors / diagnosis*
  • Lipid Metabolism, Inborn Errors / enzymology
  • Lipid Metabolism, Inborn Errors / genetics
  • Lipid Metabolism, Inborn Errors / therapy*
  • Mass Spectrometry
  • Multiple Organ Failure / enzymology
  • Multiple Organ Failure / etiology*
  • Multiple Organ Failure / genetics
  • Riboflavin / administration & dosage

Substances

  • Dietary Carbohydrates
  • Dietary Fats
  • Acetylcarnitine
  • Acyl-CoA Dehydrogenases
  • Acyl-CoA Dehydrogenase
  • Carnitine
  • Riboflavin