Treatment of cyclic neutropenia with granulocyte colony-stimulating factor

N Engl J Med. 1989 May 18;320(20):1306-11. doi: 10.1056/NEJM198905183202003.


Six patients with cyclic neutropenia were treated with recombinant human granulocyte colony-stimulating factor (G-CSF) for 3 to 15 months. All had a history of recurrent aphthous stomatitis, pharyngitis, lymphadenopathy, fever, and numerous infections during periods of neutropenia. Serial blood-cell counts, findings on bone marrow examination, and signs and symptoms were evaluated before and during the daily administration of G-CSF (3 to 10 micrograms per kilogram of body weight per day), either intravenously or subcutaneously. The kinetics of labeled autologous blood neutrophils and the migration of neutrophils to skin chambers were also measured. Recombinant human G-CSF increased the mean (+/- SEM) neutrophil counts from 717 +/- 171 per microliter to 9814 +/- 2198 per microliter (P = 0.009). In five of the six patients, the cycling of blood-cell counts continued, but the length of the period decreased from 21 to 14 days. The number of days of severe neutropenia was reduced (P = 0.002). Neutrophil turnover increased almost four-fold (P = 0.005), whereas neutrophil migration to a skin chamber was normal. G-CSF therapy reduced the frequency of oropharyngeal inflammation, fever, and infections in these patients. During the first 40 months of treatment, no typical mouth ulcers or bacterial infections occurred; recurrent gingivitis improved. We conclude that G-CSF is effective for the treatment of cyclic neutropenia in humans.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Agranulocytosis / therapy*
  • Blood Cell Count
  • Child
  • Colony-Stimulating Factors / administration & dosage
  • Colony-Stimulating Factors / adverse effects
  • Colony-Stimulating Factors / therapeutic use*
  • Female
  • Granulocyte Colony-Stimulating Factor
  • Humans
  • Male
  • Neutropenia / therapy*
  • Neutrophils
  • Periodicity
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use


  • Colony-Stimulating Factors
  • Recombinant Proteins
  • Granulocyte Colony-Stimulating Factor