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Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.

Publication ,  Journal Article
Calderwood, S; Kilpatrick, L; Douglas, SD; Freedman, M; Smith-Whitley, K; Rolland, M; Kurtzberg, J
Published in: Blood
January 15, 2001

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.

Duke Scholars

Published In

Blood

DOI

ISSN

0006-4971

Publication Date

January 15, 2001

Volume

97

Issue

2

Start / End Page

376 / 382

Location

United States

Related Subject Headings

  • Splenomegaly
  • Recombinant Proteins
  • Neutrophils
  • Neutropenia
  • Myeloid Cells
  • Male
  • Lymphocyte Count
  • Infections
  • Infant
  • Immunology
 

Citation

APA
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ICMJE
MLA
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Calderwood, S., Kilpatrick, L., Douglas, S. D., Freedman, M., Smith-Whitley, K., Rolland, M., & Kurtzberg, J. (2001). Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b. Blood, 97(2), 376–382. https://doi.org/10.1182/blood.v97.2.376
Calderwood, S., L. Kilpatrick, S. D. Douglas, M. Freedman, K. Smith-Whitley, M. Rolland, and J. Kurtzberg. “Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.Blood 97, no. 2 (January 15, 2001): 376–82. https://doi.org/10.1182/blood.v97.2.376.
Calderwood S, Kilpatrick L, Douglas SD, Freedman M, Smith-Whitley K, Rolland M, et al. Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b. Blood. 2001 Jan 15;97(2):376–82.
Calderwood, S., et al. “Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.Blood, vol. 97, no. 2, Jan. 2001, pp. 376–82. Pubmed, doi:10.1182/blood.v97.2.376.
Calderwood S, Kilpatrick L, Douglas SD, Freedman M, Smith-Whitley K, Rolland M, Kurtzberg J. Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b. Blood. 2001 Jan 15;97(2):376–382.

Published In

Blood

DOI

ISSN

0006-4971

Publication Date

January 15, 2001

Volume

97

Issue

2

Start / End Page

376 / 382

Location

United States

Related Subject Headings

  • Splenomegaly
  • Recombinant Proteins
  • Neutrophils
  • Neutropenia
  • Myeloid Cells
  • Male
  • Lymphocyte Count
  • Infections
  • Infant
  • Immunology