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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
Published on: February 15, 2019
Prophylactic PEG-rhG-CSF Reduces Febrile Neutropenia in Pediatric Hematological Malignancies Compared with Daily
Xiao Zhang1, Yang Fu1, Hongsheng Wang1
1Department of Hematology, National Children's Medical Center, Children's Hospital of Fudan University, Shanghai 201102, China.
Insights
Prophylactic pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) significantly reduced febrile neutropenia (FN) incidence in pediatric cancer patients compared to daily short-acting rhG-CSF. Safety profiles were similar, though larger studies are needed.
Area of Science:
- Pediatric Hematology/Oncology
- Pharmacology
- Clinical Trials
Background:
- Febrile neutropenia (FN) is a serious complication in pediatric hematological malignancies treated with chemotherapy.
- Current treatment often involves daily recombinant human granulocyte colony-stimulating factor (rhG-CSF).
- Pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) offers a prolonged-acting alternative.
Purpose of the Study:
- To compare the efficacy and safety of single-dose PEG-rhG-CSF versus daily short-acting rhG-CSF in preventing FN in pediatric patients.
- To evaluate secondary endpoints including neutropenia duration, recovery time, and healthcare resource utilization.
- To assess the safety profile of PEG-rhG-CSF in this patient population.
Main Methods:
- A single-center, open-label, randomized controlled trial involving pediatric patients (<18 years) with leukemia or lymphoma.
- Patients received either a single dose of PEG-rhG-CSF (100 μg/kg) or daily short-acting rhG-CSF (5 μg/kg/day) post-chemotherapy.
- Primary endpoint was FN incidence; secondary endpoints included neutropenia duration, neutrophil recovery time, and adverse events.
Main Results:
- PEG-rhG-CSF significantly reduced FN incidence (59.3%) compared to daily rhG-CSF (77.7%) (p=0.046).
- Subgroup analysis showed significant FN reduction in non-Hodgkin lymphoma patients treated with PEG-rhG-CSF.
- No significant differences were found in FN duration, time to neutrophil recovery, transfusions, hospital stay, or costs. Adverse events were similar and mild.
Conclusions:
- Single-dose prophylactic PEG-rhG-CSF is effective in lowering FN incidence in pediatric hematological malignancy patients compared to daily rhG-CSF.
- The safety profile of PEG-rhG-CSF appears comparable to daily rhG-CSF.
- Larger studies are needed to confirm safety and efficacy, especially for detecting rare adverse events.
Abstract:
Background: Febrile neutropenia (FN) is a major complication of chemotherapy in pediatric hematological malignancies. This study compared the efficacy and safety of prophylactic pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) versus daily short-acting recombinant human granulocyte colony-stimulating factor (rhG-CSF). Methods: This single-center, open-label, randomized controlled trial was conducted at a tertiary children's hospital in China. Pediatric patients (<18 years) with confirmed hematological malignancies (leukemia or lymphoma) were enrolled. A total of 138 chemotherapy cycles were randomized 2:1 to receive either a single dose of PEG-rhG-CSF (100 μg/kg, n = 86 cycles) or daily short-acting rhG-CSF (5 μg/kg/day, n = 45 cycles) after chemotherapy. The primary endpoint was FN incidence. Secondary endpoints included neutropenia incidence, FN duration, time to neutrophil recovery, blood product transfusions, hospital stay, and costs. Safety was assessed by monitoring adverse events (AEs) graded according to NCI CTCAE version 4.03. Results: Baseline characteristics were comparable between groups. PEG-rhG-CSF significantly reduced the incidence of FN (59.3% vs. 77.7%, OR 0.42 (0.18-0.97), p = 0.046) compared to daily rhG-CSF. The median time to neutrophil recovery was 9.8 days (95% CI: 8.1-10.7) in the PEG-rhG-CSF group and 10.3 days (95% CI: 8.6-11.1) in the control group (p = 0.45). No significant differences were observed in FN duration, transfusions, hospital stay, or costs. Subgroup analyses showed PEG-rhG-CSF significantly reduced FN in non-Hodgkin lymphoma (57.4% vs. 83.3%, OR 0.27 (0.08-0.89), p = 0.032). A total of 12 AEs (9.9% overall, mainly bone pain) were observed, with no significant difference between groups and no infection-related deaths. Conclusions: In this single-center, open-label trial, prophylactic single-dose PEG-rhG-CSF was associated with a lower incidence of FN compared to daily short-acting rhG-CSF. Safety profiles appeared broadly similar, but the sample size was insufficient to detect rare differences.
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