Kostmann syndrome or infantile genetic agranulocytosis, part two: Understanding the underlying genetic defects in severe congenital neutropenia

Acta Paediatr. 2007 Jun;96(6):813-9. doi: 10.1111/j.1651-2227.2007.00274.x.

Abstract

Congenital neutropenia in man was first reported 50 years ago by the Swedish paediatrician Rolf Kostmann. He coined the term 'infantile genetic agranulocytosis' for this condition, which is now known as Kostmann syndrome. Recent studies have revealed mutations in ELA-2, encoding the neutrophil granule protease, neutrophil elastase, in autosomal dominant neutropenia, and mutations in HAX-1, encoding an anti-apoptotic protein, in autosomal recessive neutropenia.

Conclusion: Future studies should aim to clarify the mechanisms underlying the evolution of secondary malignancies in these patients.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adaptor Proteins, Signal Transducing
  • Apoptosis / genetics
  • DNA-Binding Proteins / genetics
  • Genes, Recessive
  • Granulocyte Colony-Stimulating Factor
  • Humans
  • Infant
  • Leukemia / genetics
  • Leukocyte Elastase / genetics
  • Mutation
  • Myeloid Progenitor Cells / physiology
  • Neoplasms, Second Primary / etiology
  • Neutropenia / congenital
  • Neutropenia / genetics*
  • Pedigree
  • Proteins / genetics
  • Receptors, Granulocyte Colony-Stimulating Factor / genetics
  • Syndrome
  • Transcription Factors / genetics

Substances

  • Adaptor Proteins, Signal Transducing
  • DNA-Binding Proteins
  • HAX1 protein, human
  • IGHMBP2 protein, human
  • Proteins
  • Receptors, Granulocyte Colony-Stimulating Factor
  • Transcription Factors
  • Granulocyte Colony-Stimulating Factor
  • Leukocyte Elastase