[Gaucher disease in childhood]

Rev Med Interne. 2007 Oct:28 Suppl 2:S183-6. doi: 10.1016/s0248-8663(07)78879-3.
[Article in French]

Abstract

Gaucher disease is well-known in adult patients and must be regarded as a pediatric disease, two thirds of the patients manifesting before the age of 20. Three clinical forms have been defined based on the presence of neurological involvement. Gaucher disease type 1, without neurological signs, generally begins before the five years age with splenomegaly as the main symptom. The bone crises are more frequent than in adulthood. Gaucher disease type 2 or acute neuronopathic form begins between three and six months and do not have any treatment. Type 3 or chronic neuronopathic form appears like a type 1 with progressive horizontal saccade-initiation failure and developmental delay. Onset in childhood is predictive of a severe and progressive phenotype. The presence of neurological symptoms induces important consequences for treatment, prognosis and genetic counselling.

Publication types

  • Comparative Study
  • English Abstract

MeSH terms

  • Adolescent
  • Adult
  • Age Factors
  • Analgesics / therapeutic use
  • Child
  • Child, Preschool
  • Diphosphonates / therapeutic use
  • Disease Progression
  • Electroencephalography
  • Gaucher Disease* / classification
  • Gaucher Disease* / diagnosis
  • Gaucher Disease* / epidemiology
  • Gaucher Disease* / genetics
  • Gaucher Disease* / therapy
  • Genetic Counseling
  • Glucosylceramidase / administration & dosage
  • Glucosylceramidase / therapeutic use
  • Humans
  • Incidence
  • Infant
  • Infant, Newborn
  • Phenotype
  • Prognosis
  • Splenomegaly / etiology
  • Time Factors

Substances

  • Analgesics
  • Diphosphonates
  • Glucosylceramidase