Erdheim-Chester disease

Klin Onkol. 2021 Fall;34(6):434-439. doi: 10.48095/ccko2021434.

Abstract

Erdheim-Chester disease is a rare inflammatory myeloid clonal disease which is classified into histiocytoses. It is characterized by excessive production and accumulation of foamy histiocytes and Touton giant cells in various tissues and organs. Foamy histiocytes and Touton giant cells produce proinflammatory cytokines and chemokines and contain somatic mutations in genes activating the MAPK/ERK signaling pathway, but also in genes activating the PI3K/AKT signaling pathway. BRAFV600E is the most common somatic mutation. Furthermore, somatic mutations in the MAP2K1, KRAS, NRAS, ARAF or PIK3CA genes are abundant. Erdheim-Chester dis-ease is a multisystemic disease in which any organ can be affected, especially the long bones of the lower extremities, but also the cardiovascular system, retroperitoneum, endocrine system, central nervous system, lungs, skin or orbit. The dia-gnosis is difficult because of the various manifestations of this disease. The disease occurs mainly in adults and is more common in men than in women. Targeted treatment by kinase inhibitors, interferon a, cytokine blockers or cladribine is used for the treatment.

Keywords: Erdheim-Chester disease; Prognosis; heart failure; histiocytosis; incidence; survival.

Publication types

  • Review

MeSH terms

  • Cladribine / therapeutic use
  • Erdheim-Chester Disease* / diagnosis
  • Erdheim-Chester Disease* / drug therapy
  • Erdheim-Chester Disease* / epidemiology
  • Erdheim-Chester Disease* / genetics
  • Humans
  • Interferon-alpha / therapeutic use
  • Interleukin 1 Receptor Antagonist Protein / therapeutic use
  • MTOR Inhibitors / therapeutic use
  • Protein Kinase Inhibitors / therapeutic use

Substances

  • Interferon-alpha
  • Interleukin 1 Receptor Antagonist Protein
  • MTOR Inhibitors
  • Protein Kinase Inhibitors
  • Cladribine