Inaba, H., Cao, X., Pounds, S., Pui, C.H., Rubnitz, J.E., Ribeiro, R.C., & Razzouk, B.I. (2011). Randomized trial of two dosages of prophylactic granulocyte–colony-stimulating factor after induction chemotherapy in pediatric acute myeloid leukemia. Cancer, 117, 1313–1320.

The purpose of the study was to compare effects of two different doses of G-CSF in pediatric patients receiving high-dose chemotherapy.

Patients receiving induction chemotherapy were randomly assigned to receive either 5 mcg/kg or 10 mc/kg G-CSF daily after the first and second chemotherapy induction courses. Differences in number of neutropenic days, hospital days, number of febrile neutropenic episodes, episodes of infection, use of IV antibiotics, antifungal therapy courses, number of transfusions, cost of supportive care, and estimates of event-free survival.

• 46 total participants
• Median age was 9.03 years, with a range of 0.05–21 years.
• Females made up 53% of the sample; males made up 47% of the sample
• All participants had previously untreated AML or myelodyplastic syndrome and were receiving induction chemotherapy.
• Specific chemotherapy protocol used is described.
   

Single-site inpatient location

The phase of care was active antitumor treatment.

The application was for pediatrics.

Double-blind, randomized, controlled trial

• NCI Common Toxicity Criteria [v.2.0]
• Neutropenia was defined as a neutrophil count of less than 0.5 x 10⁹/L
• Event-free survival was defined as time between randomization and disease recurrence, death, secondary malignancy, or last follow-up.
   

No significant differences were noted between study groups in number of neutropenic days, episodes of febrile neutropenia, days of hospitalization, episode of antibiotic and antifungal therapy, transfusions, or cost of supportive care. There was no difference between groups in proportion of complete responses, or estimates of event-free and overall survival.

No difference was noted in measured outcomes between groups of patients treated with two different doses of G-CSF.

• Small sample (less than 100 participants)
• It is unclear if patients were given prophylactic antibiotics or antifungal therapy, which would have affected related outcomes. Sample included both patients who did and did not receive allogeneic stem cell transplantation.

Findings suggest that lower daily doses of prophylactic G-CSF can be as effective as higher doses in pediatric patients during induction chemotherapy. Dosage and timing of prophylactic G-CSF is not fully clear.