Effects of flow-dose enzyme replacement therapy in bones in Gaucher disease patients with severe skeletal involvement

Deborah Elstein, Irith Hadas-Halpern, Menachem Itzchaki, Amnon Lahad, Ayala Abrahamov, Ari Zimran*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

58 Scopus citations

Abstract

Gaucher disease, the most common glycolipid storage disorder, is characterized by hepatosplenomegaly and skeletal involvement. Enzyme replacement therapy in both the high- and low-dose regimens has been shown to be effective in reducing the organomegaly and improving the hematological parameters of symptomatic patients with type I disease. Herein we report subjective and objective responses of bone-related complications after 2 to 4 years of low-dose enzyme replacement therapy in 14 adult type I patients with severe skeletal involvement pre-treatment. We discuss our results relative to those reported in patients on the high-dose regimen, as well as with reference to a single patient who developed new avascular necrosis despite objective improvement in radiological studies.

Original languageAmerican English
Pages (from-to)104-111
Number of pages8
JournalBlood Cells, Molecules, and Diseases
Volume22
Issue number2
DOIs
StatePublished - Aug 1996
Externally publishedYes

Keywords

  • Gaucher disease
  • alglucerase
  • avascular necrosis
  • imiglucerase

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