When one disease is not enough: succinyl-CoA: 3-oxoacid coenzyme A transferase (SCOT) deficiency due to a novel mutation in OXCT1 in an infant with known phenylketonuria

J Pediatr Endocrinol Metab. 2017 Oct 26;30(10):1121-1124. doi: 10.1515/jpem-2017-0177.

Abstract

A 9-month-old Turkish girl was admitted several times within 3 months to the hospital in reduced general condition and with extreme tachypnea. The patient had been diagnosed with phenylketonuria (PKU) in newborn screening and has been treated with a low phenylalanine diet and amino acid supplements. Each time an unexplained pronounced metabolic acidosis was noted, and the child was treated with sodium-bicarbonate and glucose-electrolyte infusions. The acidosis with only slightly abnormal glucose, normal lactate levels and pronounced ketonuria suggested a defect in ketone body utilization. Succinyl-CoA: 3-oxoacid CoA transferase (SCOT) enzyme activity was low in patient's fibroblasts. Mutation analysis of the corresponding OXCT1 gene revealed that the patient was a homozygous carrier of the mutation c.1523T>C (p.V508A). We conclude that SCOT deficiency should be considered in the differential diagnosis in patients with recurrent metabolic acidotic episodes, even if they are already known to have a metabolic disease unrelated to this.

Keywords: ketolysis; metabolic acidosis; phenylketonuria.

Publication types

  • Case Reports

MeSH terms

  • Acidosis / complications*
  • Acidosis / drug therapy
  • Acidosis / genetics
  • Coenzyme A-Transferases / deficiency*
  • Coenzyme A-Transferases / genetics
  • DNA Mutational Analysis
  • Female
  • Glucose / therapeutic use
  • Humans
  • Infant
  • Mutation
  • Phenylketonurias / complications*
  • Sodium Bicarbonate / therapeutic use

Substances

  • Sodium Bicarbonate
  • Coenzyme A-Transferases
  • 3-ketoacid CoA-transferase
  • Glucose

Supplementary concepts

  • Succinyl-CoA:3-oxoacid CoA transferase deficiency