The effect of recombinant human granulocyte-macrophage colony-stimulating factor on neutropenia and related morbidity in chronic severe neutropenia.
OBJECTIVE
To define the clinical and hematologic effects of recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) on patients with chronic severe neutropenia.


METHODS
Open-label, phase II study of rhGM-CSF.


METHODS
Inpatient hematology and surgery clinic at a university medical center.


METHODS
Four consecutive patients with chronic severe neutropenia, which in two cases was complicated by severe infection, in one case by perianal fistula, and in one case by complete rectal prolapse. Two patients had chronic idiopathic neutropenia; one patient had congenital neutropenia (myelokathexis); and one patient had autoimmune neutropenia.


METHODS
The rhGM-CSF was given intravenously or subcutaneously at starting dosages of 150 to 1000 micrograms/m2 body surface area.d for 12 to 14 consecutive days. Two patients received a second course of daily rhGM-CSF treatment after a nontreatment interval of 14 to 20 days.


RESULTS
In all four patients, the absolute neutrophil counts increased from less than 0.25 x 10(9)/L to 3.2 to 19.2 x 10(9)/L within 2 weeks of beginning rhGM-CSF therapy. Two patients had life-threatening infections that resolved during therapy. The two other patients had major ano-rectal surgery during rhGM-CSF treatment and had no postoperative infections.


CONCLUSIONS
In patients with chronic neutropenia, rhGM-CSF may increase neutrophil counts. This therapy may be a useful adjunct to antibiotic therapy for patients with infection and perioperatively for patients having anorectal surgery.
Ganser
A
A
University of Frankfurt, Federal Republic of Germany.
Ottmann
O G
OG
Erdmann
H
H
Schulz
G
G
Hoelzer
D
D
D000328:Adult; D000368:Aged; D000380:Agranulocytosis; D001003:Anal Canal / Q000601:surgery; D002908:Chronic Disease; D003115:Colony-Stimulating Factors / Q000627:therapeutic use*; D004341:Drug Evaluation; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D006133:Growth Substances / Q000627:therapeutic use*; D006801:Humans; D007239:Infections / Q000188:drug therapy / Q000209:etiology; D007958:Leukocyte Count / Q000187:drug effects; D008875:Middle Aged; D009503:Neutropenia / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000627:therapeutic use; D012007:Rectum / Q000601:surgery
D003115:Colony-Stimulating Factors; D006133:Growth Substances; D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor