PHD2 mutation and congenital erythrocytosis with paraganglioma

N Engl J Med. 2008 Dec 18;359(25):2685-92. doi: 10.1056/NEJMoa0806277.

Abstract

Prolyl hydroxylase domain (PHD) proteins play a major role in regulating the hypoxia-inducible factor (HIF) that induces expression of genes involved in angiogenesis, erythropoiesis, and cell metabolism, proliferation, and survival. Germ-line mutations in the prolyl hydroxylase domain 2 gene (PHD2) have been reported in patients with familial erythrocytosis but not in association with tumors. We describe a patient with erythrocytosis and recurrent paraganglioma who carries a newly discovered PHD2 mutation. This mutation affects PHD2 function and stabilizes HIF-alpha proteins. In addition, we demonstrate loss of heterozygosity of PHD2 in the tumor, suggesting that PHD2 could be a tumor-suppressor gene.

Publication types

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

MeSH terms

  • Adult
  • Female
  • Germ-Line Mutation*
  • Hemochromatosis Protein
  • Histocompatibility Antigens Class I / genetics
  • Homozygote
  • Humans
  • Hypoxia-Inducible Factor-Proline Dioxygenases
  • Loss of Heterozygosity*
  • Male
  • Mediastinal Neoplasms / genetics*
  • Membrane Proteins / genetics
  • Neoplasms, Second Primary / genetics
  • Paraganglioma / genetics*
  • Pedigree
  • Polycythemia / congenital
  • Polycythemia / diagnosis
  • Polycythemia / genetics*
  • Procollagen-Proline Dioxygenase / genetics*
  • Procollagen-Proline Dioxygenase / metabolism
  • Sequence Analysis, DNA

Substances

  • HFE protein, human
  • Hemochromatosis Protein
  • Histocompatibility Antigens Class I
  • Membrane Proteins
  • EGLN1 protein, human
  • Procollagen-Proline Dioxygenase
  • Hypoxia-Inducible Factor-Proline Dioxygenases