Improvement of neutropenia and neutrophil dysfunction by granulocyte colony-stimulating factor in a patient with glycogen storage disease type Ib
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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 wordsGlycogen storage disease type Ib Neutropenia Granulocyte colony-stimulating factor
absolute neutrophil count
granulocyte colony-stimulating factor
glycogen storage disease Ib
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- 1.Ambruso DR, McCabe ERB, Anderson D, Beaudet A, Ballas LM, Brandt IK, Brown B, Coleman R, Dunger DB, Falletta JM, Friedman HS, Haymond MW, Keating JP, Kinney TR, Leonard JV, Mahoney DH Jr, Matalon R, Roe TF, Simmons P, Slonim AE (1985) Infectious and bleeding complications in patients with glycogenosis Ib. Am J Dis Child 139:691–697PubMedGoogle Scholar
- 4.Ishiguro A, Nakahata T, Koike K, Yoshida H, Shimbo T, Komiyama A (1991) Induction of granulocyte and granulocytemacrophage colony-stimulating factors from human monocytes stimulated by Fc fragments of human IgG. Br J Haematol 79:14–21Google Scholar
- 6.Metcalf D (1989) Haemopoietic growth factors 2: Clinical applications. Lancet I:885–887Google Scholar
- 9.Schroten H, Roesler J, Breidenbach T, Wendel U, Elsner J, Schweitzer S, Zeidler C, Burdach S, Lohmann-Matthes ML, Wahn V, Welte K (1991) Granulocyte and granulocyte-macrophage colony-stimulating factors for treatment of neutropenia in glycogen storage disease type Ib. J Pediatr 119:748–754PubMedGoogle Scholar
- 10.Schroten H, Wendel U, Burdach S, Roesler J, Breidenbach T, Schweitzer S, Zeidler C, Welte K (1991) Colony-stimulating factors for neutropenia in glycogen storage disease Ib. Lancet I:736–737Google Scholar
- 11.Souza LM, Boone TC, Gabrilove J, Lai PH, Zsebo KM, Murdock DC, Chazin VR, Bruszewski J, Lu H, Chen KK, Barendt J, Platzer E, Moore MAS, Martelsmann R, Welte K (1986) Recombinant human granulocyte colony-stimulating factor: effects on normal and leukemic myeloid cells. Science 232:61–65PubMedGoogle Scholar