Genetic defects in cytolysis in macrophage activation syndrome

Curr Rheumatol Rep. 2014;16(9):439. doi: 10.1007/s11926-014-0439-2.

Abstract

Macrophage activation syndrome (MAS), typically presenting beyond the first year of life, is an often lethal cousin of familial hemophagocytic lymphohistiocytosis (fHLH). Defects in natural killer (NK) cell and CD8 T cell cytotoxicity result in a pro-inflammatory cytokine storm, cytopenia, coagulopathy, and multi-organ system dysfunction. MAS can occur in association with infections (herpes viruses), cancer (leukemia), immune deficient states (post-transplantation), and in autoimmune (systemic lupus erythematosus) and autoinflammatory conditions (systemic juvenile idiopathic arthritis). The distinction between fHLH, the result of homozygous defects in cytolytic pathway genes, and MAS is becoming blurred with the identification of single or multiple mutations in the same cytolytic pathway genes in patients with later onset MAS. Here, we review the literature and present novel cytolytic pathway gene mutations identified in children with MAS. We study the inhibitory effect of one these novel mutations on NK cell function to suggest a direct link between fHLH and MAS.

Publication types

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

MeSH terms

  • Arthritis, Juvenile / genetics
  • Arthritis, Juvenile / immunology
  • CD8-Positive T-Lymphocytes / immunology
  • Cytotoxicity, Immunologic / genetics*
  • Humans
  • Killer Cells, Natural / immunology
  • Lymphohistiocytosis, Hemophagocytic / genetics
  • Lymphohistiocytosis, Hemophagocytic / immunology
  • Macrophage Activation Syndrome / genetics*
  • Macrophage Activation Syndrome / immunology
  • Munc18 Proteins / genetics
  • Mutation*

Substances

  • Munc18 Proteins
  • STXBP2 protein, human