title: A randomized trial of granulocyte-macrophage colony-stimulating factor in neonates with sepsis and neutropenia.
abstract: OBJECTIVE
  To determine whether adjunctive therapy with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) could reverse sepsis-associated neonatal neutropenia and improve neonatal survival and to assess its safety compared with conventional therapy in a control group.
  
  
  METHODS
  This prospective, randomized, controlled trial was performed in 60 infants with neutropenia and clinical signs of sepsis. A subcutaneous injection of rhGM-CSF (5 microgram/kg/day) was administered to 30 of the patients for 7 consecutive days. Hematologic parameters (absolute neutrophil, eosinophil, monocyte, lymphocyte counts, and platelet number) and outcome were compared with 30 conventionally treated (control) patients.
  
  
  RESULTS
  Twenty-five patients from the GM-CSF-treated group and 24 from the conventionally treated group had early-onset sepsis (</=3 days' postnatal age), and the other 11 patients had late-onset sepsis (>3 days' postnatal age). There was no difference between groups in terms of birth weight; gestational age; gender; maturity; maternal age; and incidence of prolonged rupture of membranes, maternal hypertension, or severity of sepsis. All neonates tolerated GM-CSF well with no adverse reactions. The absolute neutrophil count on day 7 was significantly increased in the GM-CSF-treated group compared with the conventionally treated group: 8088 +/- 2822/mm(3) versus 2757 +/- 823/mm(3). The mean platelet count was significantly higher on days 14 in the GM-CSF-treated group compared with conventionally treated group: 266 867 +/- 55 102/mm(3) versus 229 200 +/- 52 317/mm(3). Hematologic parameters were otherwise similar between groups before treatment and on day 28. Twenty-seven neonates in the rh-GMCSF group and 21 in the control group survived to hospital discharge. The mortality rate in the rhGM-CSF group (10%) was significantly lower than in the conventionally treated group (30%).
  
  
  CONCLUSIONS
  Treatment with rhGM-CSF is associated with an increase in absolute neutrophil, eosinophil, monocyte, lymphocyte, and platelet counts and decreased mortality in critically ill septic neutropenic neonates. These results suggest that rhGM-CSF may be effective in the treatment of neonatal sepsis with neutropenia, and further randomized trials are needed to confirm its beneficial effects.
authors.affiliation: Department of Pediatrics, Dicle Medical School, Diyarbakir, Turkey. dryaramis@hotmail.com
authors.forename: K
authors.initials: K
authors.lastname: Bilgin
authors.forename: A
authors.initials: A
authors.lastname: Yaramiş
authors.forename: K
authors.initials: K
authors.lastname: Haspolat
authors.forename: M A
authors.initials: MA
authors.lastname: Taş
authors.forename: S
authors.initials: S
authors.lastname: Günbey
authors.forename: O
authors.initials: O
authors.lastname: Derman
mesh_terms: D001424:Bacterial Infections / Q000097:blood / Q000150:complications* / Q000401:mortality; D004804:Eosinophils / Q000187:drug effects; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D007231:Infant, Newborn; D007902:Length of Stay; D007958:Leukocyte Count; D018655:Lymphocyte Count; D008297:Male; D009503:Neutropenia / Q000097:blood / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D009504:Neutrophils / Q000187:drug effects; D011446:Prospective Studies; D011994:Recombinant Proteins; D015996:Survival Rate
chemical_list: D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor