Holocarboxylase synthetase deficiency: report of one case

Acta Paediatr Taiwan. 2006 Nov-Dec;47(6):309-11.

Abstract

Holocarboxylase synthetase (HCS) is an enzyme that catalyzes biotin incorporation into carboxylases, and its deficiency causes biotin-responsive multiple carboxylase deficiency. We report a patient who had his first episode at 32 months of age. The main clinical findings were a characteristic rash, projectile vomiting, progressive consciousness loss, organophosphate order, and hypotension. Laboratory examinations showed metabolic acidosis with ketolactic acidosis, hyperammonemia, and urine organic acid profile suggestive of a biotin utilization abnormality consistent with multiple carboxylase deficiency. Nucleotide sequence analysis of the biotinidase gene of the patient revealed negative finding, however, analysis of HCS gene found a homozygous 1809C->T (R508W) mutation. R508W is a rare mutation in Taiwanese HCS deficiency patients, which is associated with the late-onset phenotype. The patient responded dramatically to biotin, and has remained normal growth and development during more than three years of follow-up. Therefore, a high index of suspicion for timely diagnosis and treatment could prevent severe complications.

Publication types

  • Case Reports

MeSH terms

  • Biotin / therapeutic use
  • Carbon-Nitrogen Ligases / genetics*
  • Child, Preschool
  • Holocarboxylase Synthetase Deficiency / drug therapy
  • Holocarboxylase Synthetase Deficiency / genetics*
  • Humans
  • Male
  • Mutation*

Substances

  • Biotin
  • Carbon-Nitrogen Ligases
  • holocarboxylase synthetases