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Improvement of neutropenia and neutrophil dysfunction by granulocyte colony-stimulating factor in a patient with

A Ishiguro1, T Nakahata, T Shimbo

  • 1Department of Paediatrics, Shinshu University School of Medicine, Matsumoto, Japan.

European Journal of Pediatrics
|January 1, 1993
PubMed

Insights

Granulocyte colony-stimulating factor (G-CSF) effectively treats glycogen storage disease type Ib (GSD Ib). This therapy increases neutrophil counts, reduces infections, and improves quality of life in GSD Ib patients.

Area of Science:

  • Biochemistry
  • Immunology
  • Pediatrics

Background:

  • Glycogen storage disease type Ib (GSD Ib) is characterized by neutropenia and impaired neutrophil function, leading to recurrent bacterial infections.
  • Neutrophil dysfunction in GSD Ib compromises the immune system's ability to fight off infections.

Observation:

  • A 9-year-old boy with GSD Ib received subcutaneous recombinant human granulocyte colony-stimulating factor (G-CSF).
  • Initial daily G-CSF injections of 100 mcg/m² significantly elevated absolute neutrophil counts and enhanced neutrophil mobility.

Findings:

  • Long-term treatment (22 months) with daily or twice-weekly G-CSF (70-100 mcg/m²) maintained elevated neutrophil counts.
  • This G-CSF regimen markedly reduced infection frequency and hospitalizations compared to untreated periods.
  • Weekly G-CSF injections at 70 mcg/m² showed less efficacy.

Implications:

  • Daily and twice-weekly G-CSF administration is a safe and effective long-term therapy for GSD Ib.
  • G-CSF represents a promising therapeutic option for neutrophilic impairment resulting from metabolic disorders.
  • This approach can significantly improve the quality of life for patients with GSD Ib by mitigating infection risks.

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