Mevalonate kinase deficiency: Evidence for a phenotypic continuum

Neurology. 2004 Mar 23;62(6):994-7. doi: 10.1212/01.wnl.0000115390.33405.f7.

Abstract

Both mevalonic aciduria, characterized by psychomotor retardation, cerebellar ataxia, recurrent fever attacks, and death in early childhood, and hyper-immunoglobulin D (hyper-IgD) syndrome, with recurrent fever attacks without neurologic symptoms, are caused by a functional deficiency of mevalonate kinase. In a systematic review of known mevalonate kinase-deficient patients, the authors identified five adults with phenotypic overlap between these two syndromes, which argues for a continuous spectrum of disease. Mevalonate kinase deficiency should be considered in adult patients with fitting neurologic symptoms, with or without periodic fever attacks.

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Enzyme Activation / genetics
  • Exanthema / genetics*
  • Exanthema / metabolism
  • Female
  • Fever / genetics*
  • Fever / metabolism
  • Humans
  • Immunoglobulin D / metabolism*
  • Infant
  • Lymphatic Diseases / genetics*
  • Lymphatic Diseases / metabolism
  • Male
  • Mevalonic Acid / metabolism
  • Mevalonic Acid / urine
  • Middle Aged
  • Mutation
  • Periodicity
  • Phenotype*
  • Phosphotransferases (Alcohol Group Acceptor) / deficiency*
  • Phosphotransferases (Alcohol Group Acceptor) / genetics
  • Recurrence
  • Registries / statistics & numerical data
  • Syndrome

Substances

  • Immunoglobulin D
  • Phosphotransferases (Alcohol Group Acceptor)
  • mevalonate kinase
  • Mevalonic Acid