The natural course of Gaucher disease in The Netherlands: implications for monitoring of disease manifestations

J Inherit Metab Dis. 2000 Feb;23(1):77-82. doi: 10.1023/a:1005655031239.

Abstract

This retrospective study in 20 untreated type I Gaucher disease patients shows that in Dutch patients clinical manifestations of Gaucher disease type I are progressive in the majority of patients, children as well as adults. This is in contrast with studies among Jewish patients. Our results emphasize the need for a regular follow-up to enable timely initiation of enzyme therapy.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Gaucher Disease / complications*
  • Humans
  • Male
  • Middle Aged
  • Retrospective Studies