De-novo mutation and sporadic presentation of acute intermittent porphyria

Lancet. 1995 Oct 14;346(8981):1007-8. doi: 10.1016/s0140-6736(95)91692-x.

Abstract

Acute intermittent porphyria (AIP) is a low-penetrant autosomal dominant disorder characterised by life-threatening neurovisceral attacks, often precipitated by drugs. Prognosis is improved by presymptomatic diagnosis and counselling. We found that 29 of 103 (28%) unrelated patients presented as sporadic cases. One patient had a de-novo mutation. The same mutation has arisen de novo at least three times in 12% of British AIP families. About 3% (95% CI 1-8%) of all AIP index cases may have de-novo mutations. These findings emphasise the high frequency of sporadic presentation of AIP and indicate the limitations of presymptomatic diagnosis as an aid to management.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • DNA Mutational Analysis
  • Female
  • Germ-Line Mutation
  • Humans
  • Hydroxymethylbilane Synthase / genetics
  • Molecular Sequence Data
  • Mutation*
  • Pedigree
  • Porphyria, Acute Intermittent / genetics*
  • Prognosis

Substances

  • Hydroxymethylbilane Synthase