Two families with compound heterozygosity for adenine phosphoribosyltransferase deficiency

Pediatr Nephrol. 2010 Jun;25(6):1173-6. doi: 10.1007/s00467-009-1430-4. Epub 2010 Jan 26.

Abstract

Adenine phosphoribosyltransferase deficiency is a disorder in which 2,8-dihydroxyadenine (2,8-DHA) crystalluria is caused by a congenital deficiency in the enzyme adenine phosphoribosyltransferase (APRT). In most cases, APRT deficiency is caused by autosomal recessive inheritance of a homozygote of the mutant gene APRT*Q0 or APRT*J, but there are also some cases in which the disorder is caused by the compound heterozygote APRT*Q0 and APRT*J. In the patients described here, brown round crystals were found in their urinary sediment. Crystalluria was the first sign of APRT deficiency, thereafter confirmed by genetic screening for APRT*/Q0 and APRT*. We performed genetic screening for APRT*Q0 and APRT*J in two families and diagnosed three cases of APRT*Q0 /APRT*J compound heterozygote-type APRT deficiency. Genetic screening for APRT*Q0 and APRT*J of family members is effective for early diagnosis and early treatment for family members.

Publication types

  • Case Reports

MeSH terms

  • Adenine Phosphoribosyltransferase / deficiency*
  • Adenine Phosphoribosyltransferase / genetics*
  • Child, Preschool
  • Female
  • Heterozygote*
  • Humans
  • Infant
  • Kidney Failure, Chronic / genetics
  • Male
  • Mutation
  • Pedigree

Substances

  • Adenine Phosphoribosyltransferase