A common mutation is associated with a mild, potentially asymptomatic phenotype in patients with isovaleric acidemia diagnosed by newborn screening

Am J Hum Genet. 2004 Dec;75(6):1136-42. doi: 10.1086/426318. Epub 2004 Oct 14.

Abstract

Isovaleric acidemia (IVA) is an inborn error of leucine metabolism that can cause significant morbidity and mortality. Since the implementation, in many states and countries, of newborn screening (NBS) by tandem mass spectrometry, IVA can now be diagnosed presymptomatically. Molecular genetic analysis of the IVD gene for 19 subjects whose condition was detected through NBS led to the identification of one recurring mutation, 932C-->T (A282V), in 47% of mutant alleles. Surprisingly, family studies identified six healthy older siblings with identical genotype and biochemical evidence of IVA. Our findings indicate the frequent occurrence of a novel mild and potentially asymptomatic phenotype of IVA. This has significant consequences for patient management and counseling.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Algorithms
  • DNA Primers
  • Genotype
  • Hemiterpenes
  • Humans
  • Infant, Newborn
  • Isovaleryl-CoA Dehydrogenase
  • Leucine / metabolism*
  • Mass Spectrometry
  • Metabolism, Inborn Errors / diagnosis
  • Metabolism, Inborn Errors / genetics*
  • Mutation / genetics*
  • Neonatal Screening
  • Oxidoreductases Acting on CH-CH Group Donors / deficiency*
  • Oxidoreductases Acting on CH-CH Group Donors / genetics*
  • Pentanoic Acids
  • Restriction Mapping

Substances

  • DNA Primers
  • Hemiterpenes
  • Pentanoic Acids
  • isovaleric acid
  • Oxidoreductases Acting on CH-CH Group Donors
  • Isovaleryl-CoA Dehydrogenase
  • Leucine