Familial and acquired hemophagocytic lymphohistiocytosis

GE Janka - Annual review of medicine, 2012 - annualreviews.org
GE Janka
Annual review of medicine, 2012annualreviews.org
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening syndrome in which an
uncontrolled and ineffective immune response, triggered in most cases by infectious agents,
leads to severe hyperinflammation. Familial forms of HLH (FHL), which are increasingly
found also in adolescents and adults, are due to genetic defects leading to impaired function
of natural killer cells and cytotoxic T cells. These mutations occur either in the perforin gene
or in genes important for the exocytosis of cytotoxic granules. Cytotoxic granules contain …
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening syndrome in which an uncontrolled and ineffective immune response, triggered in most cases by infectious agents, leads to severe hyperinflammation. Familial forms of HLH (FHL), which are increasingly found also in adolescents and adults, are due to genetic defects leading to impaired function of natural killer cells and cytotoxic T cells. These mutations occur either in the perforin gene or in genes important for the exocytosis of cytotoxic granules. Cytotoxic granules contain perforin and granzymes, which induce apoptosis upon entering (infected) target cells. Additionally, perforin is important for the downregulation of the immune response. Acquired forms of HLH are encountered in association with (usually) viral infections, autoinflammatory/autoimmune diseases, malignant diseases, and acquired immune deficiency states (e.g., after organ transplantation). Treatment of HLH includes immune-suppressive and immune-modulatory agents, cytostatic drugs, and biological response modifiers. For patients with FHL, stem cell transplantation is indicated and can be curative.
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