title: Effect of granulocyte-colony stimulating factor in glycogen storage disease type Ib.
abstract: Five children with glycogen storage disease type Ib (glycogenosis Ib), a metabolic defect associated with neutropenia and impairment of neutrophil function, were treated with granulocyte colony stimulating factor, a haemopoietic growth factor that induces a significant increase in polymorphonuclear leucocyte count in vivo. Recurrent bacterial or fungal infections were recorded in the three patients who had very low polymorphonuclear leucocyte counts. Experience at this centre indicates that the absolute number of polymorphonuclear leucocytes is more important than the alteration of their metabolic function in determining the susceptibility of the patients to infection. Granulocyte colony stimulating factor was effective at increasing polymorphonuclear leucocyte numbers (and restored function to some extent) in patients with glycogenosis Ib. This drug might be beneficial in cases of severe infection, in glycogenesis Ib patients with neutropenia.
authors.affiliation: 5th Department of Pediatrics, University of Milan, Italy.
authors.forename: G V
authors.initials: GV
authors.lastname: Zuccotti
authors.forename: R
authors.initials: R
authors.lastname: Longhi
authors.forename: P
authors.initials: P
authors.lastname: Flumine
authors.forename: A
authors.initials: A
authors.lastname: Stangalini
authors.forename: E
authors.initials: E
authors.lastname: Riva
mesh_terms: D001424:Bacterial Infections / Q000097:blood / Q000150:complications; D002634:Chemotaxis, Leukocyte / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D005260:Female; D006009:Glycogen Storage Disease Type II / Q000097:blood* / Q000188:drug therapy*; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D007958:Leukocyte Count / Q000187:drug effects; D008297:Male; D009181:Mycoses / Q000097:blood / Q000150:complications; D009504:Neutrophils / Q000187:drug effects / Q000378:metabolism / Q000502:physiology*; D010587:Phagocytosis / Q000187:drug effects
chemical_list: D016179:Granulocyte Colony-Stimulating Factor