title: Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.
abstract: The purpose of this study was to evaluate the efficacy and toxicity of recombinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b. Thirteen patients with neutropenia and/or neutrophil dysfunction secondary to GSD type 1b were treated with rhG-CSF. The effects of therapy on neutrophil numbers and in vitro neutrophil function and on bone marrow cellularity and morphology were studied. The clinical status of the patients and the occurrence of adverse events associated with rhG-CSF use were monitored. Use of rhG-CSF therapy was associated with a significant increase in circulating neutrophil numbers (P <. 01) and an improvement in neutrophil function as assessed in vitro. In addition, rhG-CSF therapy produced a significant increase in marrow cellularity and an increase in myeloid:erythroid (M:E) ratio, indicating stimulation of granulopoeisis. No adverse effects on marrow function were noted; in particular, no myelodysplasia or marrow exhaustion was seen. Use of rhG-CSF therapy was associated with objective and subjective improvements in infection-related morbidity. The therapy was well tolerated, although all patients developed splenomegaly, and 5 patients developed mild hypersplenism that did not require any specific treatment. rhG-CSF therapy is efficacious in the management of neutropenia and neutrophil dysfunction associated with GSD type 1b. Patients on this therapy need to be monitored for hypersplenism. Continued follow-up will be necessary to confirm long-term safety; however, no significant short-term toxicity was noted.
authors.affiliation: City of Hope/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ, USA. scalderwood@coh.org
authors.forename: S
authors.initials: S
authors.lastname: Calderwood
authors.forename: L
authors.initials: L
authors.lastname: Kilpatrick
authors.forename: S D
authors.initials: SD
authors.lastname: Douglas
authors.forename: M
authors.initials: M
authors.lastname: Freedman
authors.forename: K
authors.initials: K
authors.lastname: Smith-Whitley
authors.forename: M
authors.initials: M
authors.lastname: Rolland
authors.forename: J
authors.initials: J
authors.lastname: Kurtzberg
mesh_terms: D000293:Adolescent; D000328:Adult; D001854:Bone Marrow Cells / Q000166:cytology / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D004341:Drug Evaluation; D005260:Female; D005953:Glycogen Storage Disease Type I / Q000150:complications* / Q000188:drug therapy / Q000473:pathology; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage* / Q000592:standards / Q000633:toxicity; D006801:Humans; D007223:Infant; D007239:Infections / Q000209:etiology; D018655:Lymphocyte Count; D008297:Male; D022423:Myeloid Cells / Q000187:drug effects; D009503:Neutropenia / Q000188:drug therapy* / Q000209:etiology; D009504:Neutrophils / Q000187:drug effects / Q000473:pathology; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000592:standards / Q000633:toxicity; D013163:Splenomegaly / Q000139:chemically induced
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor