Granulocyte colony-stimulating factor in glycogen storage disease type 1b. Results of the European Study on Glycogen Storage Disease Type 1

Patients with glycogen storage disease type 1b (GSD-1b) have neutropenia and neutrophil dysfunction that predispose to frequent infections and inflammatory bowel disease (IBD), for which granulocyte colony-stimulating factor (GCSF) is given. To investigate the use and the value of GCSF treatment in...

Full description

Saved in:
Bibliographic Details
Published in:European journal of pediatrics 2002-10, Vol.161 Suppl 1 (1), p.S83-S87
Main Authors: Visser, Gepke, Rake, Jan Peter, Labrune, Philippe, Leonard, James V, Moses, Shimon, Ullrich, Kurt, Wendel, Udo, Groenier, Klaas H, Smit, G Peter A
Format: Article
Language:English
Subjects:
Child
Female
Glycogen Storage Disease Type I - complications
Glycogen Storage Disease Type I - drug therapy
Granulocyte Colony-Stimulating Factor - administration & dosage
Granulocyte Colony-Stimulating Factor - therapeutic use
Humans
Male
Neutropenia - drug therapy
Neutropenia - etiology
Retrospective Studies
Treatment Outcome
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Patients with glycogen storage disease type 1b (GSD-1b) have neutropenia and neutrophil dysfunction that predispose to frequent infections and inflammatory bowel disease (IBD), for which granulocyte colony-stimulating factor (GCSF) is given. To investigate the use and the value of GCSF treatment in GSD-1b, a retrospective registry of GSD-1 patients born between 1960 and 1995 in 12 European countries was established. Included were 57 GSD-1b patients. Unglycosylated GCSF was given to 18 patients, median age of starting therapy was 8 years, longest duration of therapy 7 years. Dose varied between 2-10 micro g/kg, with a frequency from daily to twice per week. Neutropenia (defined as an absolute neutrophil count
ISSN:0340-6199
1432-1076
DOI:10.1007/bf02680001