Recombinant human granulocyte-colony-stimulating factor in the treatment of patients with chronic benign granulocytopenia and congenital agranulocytosis (Kostmann's syndrome)

Acta Paediatr. 1992 Feb;81(2):133-6. doi: 10.1111/j.1651-2227.1992.tb12188.x.

Abstract

Seven patients with chronic benign granulocytopenia and nine patients with congenital agranulocytosis, received consecutive seven-day courses of recombinant human granulocyte-colony stimulating factor at a starting dose of 50 micrograms/m2/day, subcutaneously. If there was no response the doses were increased to 300 micrograms/m2. All patients with chronic benign granulocytopenia responded rapidly at the minimum dose within 1-3 days after administration. By contrast, only three of the nine patients with congenital agranulocytosis responded within 1-7 days at this dose. Four patients with congenital agranulocytosis showed a response between days 7-19 at a dose of granulocyte-colony-stimulating factor 100-200 micrograms/m2 but in the remaining two cases no response was obtained. The administration of granulocyte-colony-stimulating factor was shown to be safe and effective also in reducing infectious episodes in these patients. Previously it was reported that granulocyte-colony-stimulating factor 10-30 micrograms/kg/day was effective for patients with congenital agranulocytosis. These results indicate that patients with congenital agranulocytosis may require much higher doses of recombinant human granulocyte-colony-stimulating factor than patients with chronic benign granulocytopenia and that the response to ordinary doses of recombinant human granulocyte-colony-stimulating factor may be useful in differentiating between chronic benign granulocytopenia and congenital agranulocytosis.

Publication types

  • Clinical Trial
  • Comparative Study

MeSH terms

  • Adolescent
  • Agranulocytosis / blood
  • Agranulocytosis / congenital
  • Agranulocytosis / diagnosis
  • Agranulocytosis / drug therapy*
  • Child
  • Child, Preschool
  • Chronic Disease
  • Dose-Response Relationship, Drug
  • Granulocyte Colony-Stimulating Factor / administration & dosage*
  • Granulocytes / drug effects
  • Granulocytes / pathology
  • Humans
  • Infant
  • Injections, Subcutaneous
  • Leukocyte Count / drug effects
  • Neutropenia / blood
  • Neutropenia / diagnosis
  • Neutropenia / drug therapy*
  • Recombinant Proteins / administration & dosage*
  • Severity of Illness Index
  • Syndrome

Substances

  • Recombinant Proteins
  • Granulocyte Colony-Stimulating Factor