Advances in understanding the pathogenesis of primary familial and congenital polycythaemia

Br J Haematol. 2010 Mar;148(6):844-52. doi: 10.1111/j.1365-2141.2009.08069.x. Epub 2010 Jan 20.

Abstract

Primary familial and congenital polycythemia (PFCP) is an autosomal-dominant proliferative disorder characterized by erythrocytosis and hypersensitivity of erythroid progenitors to erythropoietin (Epo). Several lines of evidence suggest a causal role of truncated erythropoietin receptor (EpoR) in this disease. In this review, we discuss PFCP in the context of erythrocytosis and EpoR signalling. We focus on recent studies describing mechanisms underlying Epo-dependent EpoR down-regulation. One mechanism depends on internalization mediated through the p85 regulatory subunit of the Phosphoinositide 3-Kinase, and the other utilizes ubiquitin-based proteasomal degradation. Truncated PFCP EpoRs are not properly down-regulated upon stimulation, underscoring the importance of these mechanisms in the pathogenesis of PFCP.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Down-Regulation
  • Humans
  • Mutation
  • Polycythemia / congenital
  • Polycythemia / diagnosis
  • Polycythemia / genetics*
  • Receptors, Erythropoietin / genetics
  • Receptors, Erythropoietin / physiology
  • Signal Transduction / physiology

Substances

  • Receptors, Erythropoietin