title: Recombinant human granulocyte-colony-stimulating factor in the treatment of patients with chronic benign granulocytopenia and congenital agranulocytosis (Kostmann's syndrome).
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.
authors.affiliation: Department of Pediatrics, Children's Research Hospital, Kyoto Prefectural University of Medicine, Japan.
authors.forename: S
authors.initials: S
authors.lastname: Imashuku
authors.forename: M
authors.initials: M
authors.lastname: Tsuchida
authors.forename: M
authors.initials: M
authors.lastname: Sasaki
authors.forename: T
authors.initials: T
authors.lastname: Shimokawa
authors.forename: H
authors.initials: H
authors.lastname: Nakamura
authors.forename: T
authors.initials: T
authors.lastname: Matsuyama
authors.forename: N
authors.initials: N
authors.lastname: Taniguchi
authors.forename: M
authors.initials: M
authors.lastname: Oda
authors.forename: S
authors.initials: S
authors.lastname: Higuchi
authors.forename: K
authors.initials: K
authors.lastname: Ishimoto
mesh_terms: D000293:Adolescent; D000380:Agranulocytosis / Q000097:blood / Q000151:congenital / Q000175:diagnosis / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D004305:Dose-Response Relationship, Drug; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage*; D006098:Granulocytes / Q000187:drug effects / Q000473:pathology; D006801:Humans; D007223:Infant; D007279:Injections, Subcutaneous; D007958:Leukocyte Count / Q000187:drug effects; D009503:Neutropenia / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000008:administration & dosage*; D012720:Severity of Illness Index; D013577:Syndrome
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor