The effect of recombinant human granulocyte-macrophage colony-stimulating factor on neutropenia and related morbidity in chronic severe neutropenia.

STUDY

OBJECTIVE

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

DESIGN

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

SETTING

Inpatient hematology and surgery clinic at a university medical center.

PATIENTS: 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.

INTERVENTIONS: 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.

MEASUREMENTS AND MAIN

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.