A case-control retrospective study of the efficacy of granulocyte-colony-stimulating factor in children with neuroblastoma.

Purpose: We conducted a retrospective case-control study to examine the effect of granulocyte-colony-stimulating factor (G-CSF) on the duration of the neutrophil nadir and other clinical parameters in children with neuroblastoma.

Patients and methods: We retrospectively reviewed 85 courses of the same chemotherapy in 16 consecutive neuroblastoma patients. The first nine patients received no growth factor and the following seven patients received G-CSF. Data obtained included days of neutropenia, fever rate and duration, hospitalization rate and duration, antibiotic duration, and infection rate.

Results: Patients who received G-CSF had a significant decrease in the period of neutropenia (mean 5.4 +/- 2.6 days per course vs. 11.4 +/- 4.1 days per course in the control group; p < 0.001). There were no statistically significant differences in episodes of fever per course, rate of hospitalization per course, duration of hospitalization, or duration of antibiotic therapy. Control patients had documented infections during 16% (nine of 56) of their chemotherapy courses, whereas the patients receiving G-CSF had infections during 7% (two of 29) of their courses, but this difference was not statistically significant (p = 0.318). We calculated that a study of 220 courses in each group would be needed to have adequate power to confirm that this difference is statistically significant.

Conclusions: The administration of G-CSF in this patient population did result in fewer days of neutropenia, a finding that has been reported previously in several adult studies. However, we conclude that the clinical benefit of more rapid hematologic recovery in children remains uncertain and deserves further investigation in a large, prospective multicenter trial.