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Glycogen storage disease associated with Niemann-Pick disease: histochemical, enzymatic, and lipid analyses

T Narita1, H Nakazawa, Y Hizawa

  • 1Department of Pathology, Hirosaki University School of Medicine, Japan.

Insights

This case report details a patient with Type IA glycogen storage disease who developed multiple hepatic adenomas and Niemann-Pick disease, highlighting the complex interplay of these rare conditions.

Area of Science:

  • Hepatology
  • Genetics
  • Pathology

Background:

  • Glycogen storage disease Type IA (GSD Ia) is a rare inherited metabolic disorder.
  • Hepatic adenomas are benign liver tumors that can occur in patients with GSD Ia.
  • Niemann-Pick disease is a group of rare inherited metabolic disorders affecting lipid metabolism.

Observation:

  • A female patient diagnosed with GSD Ia at birth developed a hepatocellular adenoma at age 12.
  • At age 18, she presented with hepatomegaly, jaundice, and was diagnosed with Niemann-Pick disease.
  • Autopsy revealed a significantly enlarged liver with multiple hepatocellular adenomas and spleen with infarctions.

Findings:

  • Histological and electron microscopy confirmed hepatocellular adenomas and characteristic foamy cells of Niemann-Pick disease in multiple organs.
  • Foamy cells, indicative of lipid accumulation, were identified in the spleen, liver, bone marrow, lymph nodes, kidneys, and lungs.
  • The patient died at age 19 due to pneumonia and cardiac tamponade.

Implications:

  • This case underscores the potential for multiple hepatic adenomas and Niemann-Pick disease to co-occur in patients with GSD Ia.
  • Understanding these complex co-morbidities is crucial for comprehensive patient management and prognosis.
  • Further research may elucidate shared pathways or predispositions contributing to these concurrent conditions.

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