Molecular mechanisms of neonatal hyperinsulinism

Horm Res. 2006;66(6):289-96. doi: 10.1159/000095938. Epub 2006 Sep 26.

Abstract

Congenital hyperinsulinism (CHI), characterized by profound hypoglycaemia related to inappropriate insulin secretion, may be associated histologically with either diffuse insulin hypersecretion or focal adenomatous hyperplasia, which share a similar clinical presentation, but result from different molecular mechanisms. Whereas diffuse CHI is of autosomal recessive, or less frequently of autosomal dominant, inheritance, focal CHI is sporadic. The most common mechanism underlying CHI is dysfunction of the pancreatic ATP-sensitive potassium channel (K(+)(ATP)). The two subunits of the K(+)(ATP) channel are encoded by the sulfonylurea receptor gene (SUR1 or ABCC8) and the inward-rectifying potassium channel gene (KIR6.2 or KCNJ11), both located in the 11p15.1 region. Germ-line, paternally inherited, mutations of the SUR1 or KIR6.2 genes, together with somatic maternal haplo-insufficiency for 11p15.5, were shown to result in focal CHI. Diffuse CHI results from germ-line mutations in the SUR1 or KIR6.2 genes, but also from mutations in several other genes, namely glutamate dehydrogenase (with associated hyperammonaemia), glucokinase, short-chain L-3-hydroxyacyl-CoA dehydrogenase, and insulin receptor gene. Hyperinsulinaemic hypoglycaemia may be observed in several overlapping syndromes, such as Beckwith-Wiedemann syndrome (BWS), Perlman syndrome, and, more rarely, Sotos syndrome. Mosaic genome-wide paternal isodisomy has recently been reported in patients with clinical signs of BWS and CHI. The primary causes of CHI are genetically heterogeneous and have not yet been completely unveiled. However, secondary causes of hyperinsulinism have to be considered such as fatty acid oxidation deficiency, congenital disorders of glycosylation and factitious hypoglycaemia secondary to Munchausen by proxy syndrome.

Publication types

  • Review

MeSH terms

  • ATP-Binding Cassette Transporters / genetics
  • Chromosomes, Human, Pair 11 / genetics
  • Congenital Hyperinsulinism / genetics*
  • Congenital Hyperinsulinism / physiopathology
  • Genes, Dominant
  • Genes, Recessive
  • Glutamate Dehydrogenase / genetics
  • Humans
  • Infant
  • Infant, Newborn
  • Insulin / metabolism
  • Insulin Secretion
  • Loss of Heterozygosity
  • Potassium Channels / genetics
  • Potassium Channels, Inwardly Rectifying / genetics
  • Receptors, Drug / genetics
  • Sulfonylurea Receptors
  • Syndrome

Substances

  • ABCC8 protein, human
  • ATP-Binding Cassette Transporters
  • Insulin
  • Kir6.2 channel
  • Potassium Channels
  • Potassium Channels, Inwardly Rectifying
  • Receptors, Drug
  • Sulfonylurea Receptors
  • Glutamate Dehydrogenase