A multicenter, randomized, placebo-controlled trial of prophylactic recombinant granulocyte-colony stimulating factor in preterm neonates with neutropenia

J Pediatr. 2009 Sep;155(3):324-30.e1. doi: 10.1016/j.jpeds.2009.03.019. Epub 2009 May 24.


Objective: To test the hypothesis that prophylactic treatment of neutropenic premature neonates with recombinant granulocyte-colony stimulating factor (rG-CSF) would reduce the incidence of nosocomial infections (NIs).

Study design: A total of 25 neonatal intensive care units participated in this multicenter, randomized, double-blind, placebo-controlled trial. Premature infants of gestational age (GA) <or= 32 weeks were included if they had a peripheral blood count showing < 1500 neutrophils/mm(3) for at least 24 hours during the first 3 weeks of life. A total of 200 infants received either rG-CSF (10 microg/kg/day) or placebo for 3 days. Primary outcome was survival free of infection for 4 weeks after treatment, assessed in an intention-to-treat analysis.

Results: A total of 102 infants received rG-CSF (mean GA, 29.2 weeks), and 98 received placebo (mean GA, 29.1 weeks). Survival free of confirmed infection for 4 weeks after treatment was 74/102 in the rG-CSF group and 66/98 in the placebo group (P = .42). However, during 2 weeks, there was a significant difference between groups (86/102 vs 70/98; P = .028).

Conclusions: In this population, prophylactic rG-CSF did not significantly increase survival free of infection at 4 weeks after treatment. The transient effect observed at 2 weeks in the most immature infants should be evaluated further.

Trial registration: ClinicalTrials.gov NCT00213759.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cross Infection / prevention & control
  • Double-Blind Method
  • Female
  • Gestational Age
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Humans
  • Infant, Newborn
  • Infant, Premature
  • Infant, Premature, Diseases / diagnosis
  • Infant, Premature, Diseases / drug therapy*
  • Infant, Very Low Birth Weight
  • Leukocyte Count
  • Male
  • Neutropenia / diagnosis
  • Neutropenia / drug therapy*
  • Recombinant Proteins
  • Treatment Outcome


  • Recombinant Proteins
  • Granulocyte Colony-Stimulating Factor

Associated data

  • ClinicalTrials.gov/NCT00213759