[Gaucher's disease]

J Soc Biol. 2002;196(2):141-9.
[Article in French]

Abstract

Gaucher disease is an uncommon autosomic recessive disorder. The disease is caused by a deficiency in the activity of the lysosomal enzyme glucocerebrosidase which is responsible for the degradation of glucosylceramide, résulting from the breakdown of red and white cell-membranes. In the absence of enzyme glucosylceramide accumulates in the lysosomes of macrophages. This accumulation leads to hepatomegaly, splenomegaly with subsequent haematologic abnormalities (leucopenia, anemia, thrombopenia) and bone manifestations. Three types of Gaucher disease are described: type 1 is the most common, type 2 and 3 are associated with neurologic symptoms. Macrophages are the likely cellular source of biochemical abnormalities: elevated blood level of ferritin, angiotensin converting enzyme, immunoglobulins and haemostasis abnormalities. Lysosomal perturbations lead to increased blood level of tartrate resistant acid phosphatase and chitotriosidase. Enzyme replacement therapy is available in France since 1991. In 2002, 136 patients are treated. The efficacy is overt on the asthenia, organomegaly and haematological manifestations. Bone pains disappear or decrease in intensity, however bone complications may be irreversible justifying treatment initiations before the appearance of lesions that may lead to serious functional impairment.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Biomarkers / blood
  • Bone Diseases, Metabolic / etiology
  • Bone Diseases, Metabolic / pathology
  • Bone Marrow Transplantation
  • France
  • Gaucher Disease* / blood
  • Gaucher Disease* / classification
  • Gaucher Disease* / enzymology
  • Gaucher Disease* / epidemiology
  • Gaucher Disease* / pathology
  • Gaucher Disease* / therapy
  • Genetic Therapy
  • Glucosylceramidase / deficiency*
  • Glucosylceramidase / genetics
  • Glucosylceramidase / therapeutic use
  • Hepatomegaly / etiology
  • Hepatomegaly / pathology
  • Humans
  • Lysosomes / chemistry
  • Lysosomes / ultrastructure
  • Macrophages / chemistry
  • Macrophages / pathology
  • Splenectomy / adverse effects
  • Splenomegaly / etiology
  • Splenomegaly / pathology

Substances

  • Biomarkers
  • Glucosylceramidase