A Randomized Trial of Granulocyte-Macrophage Colony-Stimulating Factor in Neonates with Sepsis and Neutropenia. Belgin K, Yaramis A, Haspolat K, et al. Pediatrics 2001; 107: 36-41.

This prospective, randomized controlled trial was performed in 60 infants with neutropenia and clinical signs of sepsis. A subcutaneous injection of rhGM-CSF (5 mcg/kg/d) was administered to 30 of the patients on 7 consecutive days. Hematologic parameters and outcome were compared with 30 conventionally treated (control) patients. Twenty-five patients from the GM-CSF group and 24 from the control group had early-onset sepsis (< 3 days of age), and the other 11 patients had late-onset sepsis. There was no differences between groups in terms of birth weight, gestational age, gender, maturity, maternal age, incidence of prolonged rupture of membranes, maternal hypertension, or severity of sepsis. All infants tolerated GM-CSF well with no adverse reactions. The absolute neutrophil count on day 7 and the platelet count on day 14 were significantly increased in the GM-CSF-treated group compared to the control group. Twenty-seven neonates in the GM-CSF group and 21 in the control group survived to hospital discharge. The mortality rate in the GM-CSF group (10%) was sinificantly lower than that of the control group (30%).