Recombinant human granulocyte-colony-stimulating factor in the treatment of patients with chronic benign granulocytopenia and congenital agranulocytosis (Kostmann's syndrome).
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.
Imashuku
S
S
Department of Pediatrics, Children's Research Hospital, Kyoto Prefectural University of Medicine, Japan.
Tsuchida
M
M
Sasaki
M
M
Shimokawa
T
T
Nakamura
H
H
Matsuyama
T
T
Taniguchi
N
N
Oda
M
M
Higuchi
S
S
Ishimoto
K
K
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
D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor