Dihydropyrimidine dehydrogenase deficiency presenting at birth

J Inherit Metab Dis. 2005;28(5):793-6. doi: 10.1007/s10545-005-4218-0.

Abstract

Dihydropyrimidine dehydrogenase (DPD) deficiency (McKusick 274270) is a clinically heterogeneous autosomal recessive disorder of pyrimidine metabolism. DPD is the enzyme that catalyses the first and the rate-limiting step in the catabolism of uracil, thymine and the analogue 5-fluorouracil. To date, more than 30 patients have been diagnosed with a complete enzyme deficiency. Here, we describe the fifth case with a complete DPD deficiency presenting at birth with severe neurological abnormalities. The patient was homozygous for the common splice-site mutation IVS14+1G > A.

Publication types

  • Case Reports

MeSH terms

  • Age of Onset
  • Antimetabolites / metabolism
  • Binding Sites
  • Digestive System Abnormalities / etiology
  • Dihydropyrimidine Dehydrogenase Deficiency*
  • Female
  • Fluorouracil / pharmacology
  • Genes, Recessive
  • Heterozygote
  • Homozygote
  • Humans
  • Infant, Newborn
  • Muscular Diseases / etiology
  • Mutation
  • Purine-Pyrimidine Metabolism, Inborn Errors / diagnosis*
  • Thymine / chemistry
  • Thymine / urine
  • Uracil / chemistry
  • Uracil / urine

Substances

  • Antimetabolites
  • Uracil
  • Thymine
  • Fluorouracil