European Journal of Pediatrics

, Volume 152, Issue 1, pp 18–20 | Cite as

Improvement of neutropenia and neutrophil dysfunction by granulocyte colony-stimulating factor in a patient with glycogen storage disease type Ib

  • A. Ishiguro
  • T. Nakahata
  • T. Shimbo
  • Y. Amano
  • K. Yasui
  • K. Koike
  • A. Komiyama
Hematology/Oncology

Abstract

Patients with glycogen storage disease type Ib (GSD Ib) suffer from recurrent bacterial infections due to neutropenia and neutrophil dysfunction. To improve the quality of life in a 9-year-old boy with GSD Ib, we subeutaneously administered recombinant human granulocyte colony-stimulating factor (G-CSF). Daily injections of 100 μg/m2 of G-CSF significantly increased absolute neutrophil counts and augmented neutrophil mobility. The patient was then treated with 70 and 100 μg/m2 of G-CSF daily and twice-weekly. The treatment maintained absolute neutrophil counts at significantly higher levels than those without treatment for 22 months and markedly decreased the frequency of infections and the necessity for hospitalisation. No adverse effects were observed during treatment. These findings indicate that daily and twice-weekly treatment with G-CSF of long duration are safe and effective for patients with GSD Ib. G-CSF may be a useful therapeutic agent in patients with neutrophilic impairment as a consequence of a metabolic disorder.

Key words

Glycogen storage disease type Ib Neutropenia Granulocyte colony-stimulating factor 

Abbreviations

ANC

absolute neutrophil count

G-CSF

granulocyte colony-stimulating factor

GSDIb

glycogen storage disease Ib

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Copyright information

© Springer-Verlag 1993

Authors and Affiliations

  • A. Ishiguro
    • 1
    • 2
  • T. Nakahata
    • 1
  • T. Shimbo
    • 2
  • Y. Amano
    • 1
  • K. Yasui
    • 1
  • K. Koike
    • 1
  • A. Komiyama
    • 1
  1. 1.Department of PaediatricsShinshu University School of MedicineMatsumotoJapan
  2. 2.Department of Paediatrics, Mizonokuchi HospitalTeikyo University School of MedicineKawasaki 213Japan

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