Massive hepatic fibrosis in Gaucher's disease: clinico-pathological and radiological features

QJM. 2000 Apr;93(4):237-44. doi: 10.1093/qjmed/93.4.237.

Abstract

Hepatomegaly is frequent in patients with type 1 Gaucher's disease and is associated with infiltration of the liver with pathological macrophages. Most patients suffer no significant clinical consequences, but a few develop portal hypertension which may progress to parenchymal liver failure. We describe four patients with Gaucher's disease who have developed portal hypertension. We have reviewed their clinical histories and all available histological and radiological material. All had severe Gaucher's disease with multi-organ involvement, and had undergone splenectomy in childhood. Histologically, this advanced liver disease was characterized by a picture of extreme and advanced confluent fibrosis occupying the central region of the liver. This massive fibrosis is associated with characteristic radiological appearances. The liver histology in these cases is highly unusual and virtually unknown in other conditions. Our studies indicate that without specific treatment the liver disease is progressive and rapidly fatal. However, institution of enzyme replacement therapy with imiglucerase may have beneficial effects even when the condition is far advanced.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Enzyme Therapy
  • Esophageal and Gastric Varices / etiology
  • Esophageal and Gastric Varices / therapy
  • Female
  • Gaucher Disease / complications*
  • Gaucher Disease / therapy
  • Hepatomegaly / etiology
  • Hepatomegaly / pathology
  • Hepatomegaly / therapy
  • Humans
  • Hypertension, Portal / etiology*
  • Hypertension, Portal / therapy
  • Liver Cirrhosis / etiology*
  • Liver Cirrhosis / pathology
  • Liver Cirrhosis / therapy
  • Liver Function Tests
  • Magnetic Resonance Imaging
  • Male
  • Middle Aged
  • Severity of Illness Index
  • Splenectomy / adverse effects
  • Tomography, X-Ray Computed

Substances

  • Enzymes