[Persistence of pulmonary lesions in a 6-year-old boy with type I Gaucher's disease treated by alglucerase since the age of 20 months]

Arch Pediatr. 1998 Dec;5(12):1341-3. doi: 10.1016/s0929-693x(99)80053-0.
[Article in French]

Abstract

Background: Gaucher's disease is a serious disorder which becomes curable by bone marrow transplantation. Enzyme replacement therapy has permitted improvement in quality of life and has been shown to decrease the disease's progression.

Observation: A 6-year-old boy was treated by alglucerase (Ceredase) since the age of 20 months. He presented at that time a failure to thrive, pancytopenia, hepatosplenomegaly and bone involvement. The lung X-rays revealed a diffuse reticulonodular pattern. After 4 years and 5 months, there was an improvement of growth and hepatosplenomegaly, associated with hematological parameters and bone changes normalization. However, an important interstitial syndrome without clinical signs persisted.

Conclusion: In our patient it is impossible to note the difference between pulmonary disease from fibrotic origin and pulmonary involvement by Gaucher's cells accumulation.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Gaucher Disease / complications
  • Gaucher Disease / drug therapy*
  • Glucosylceramidase / administration & dosage
  • Glucosylceramidase / therapeutic use*
  • Humans
  • Lung Diseases, Interstitial* / diagnostic imaging
  • Lung Diseases, Interstitial* / etiology
  • Male
  • Radiography, Thoracic
  • Time Factors
  • Tomography, X-Ray Computed

Substances

  • alglucerase
  • Glucosylceramidase