A multicenter phase II trial of primary prophylactic PEG‐rhG‐CSF in pediatric patients with solid tumors and non‐Hodgkin lymphoma after chemotherapy: An interim analysis

Abstract Background Pegylated recombinant human granulocyte colony‐stimulating factor (PEG‐rhG‐CSF) can be used in pediatric patients. This study assessed the safety and efficacy of PEG‐rhG‐CSF as a primary prophylactic drug against neutropenia after chemotherapy in pediatric patients with solid tumors or non‐Hodgkin lymphoma (NHL). Patients and Methods This phase II study (between October 2020 and March 2022) enrolled pediatric patients with solid tumors or NHL treated with high‐intensity chemotherapy and with grade ≥3 myelosuppression for at least 14 days during chemotherapy. Prophylactic PEG‐rhG‐CSF was given at 100 μg/kg body weight (maximum total dosage of 6 mg) once 24–48 h following chemotherapy for two cycles. The primary endpoint was the incidence of PEG‐rhG‐CSF‐related adverse events (AEs). The key secondary endpoints were the rates of grade 3/4 neutropenia and febrile neutropenia (FN). Results This study included 160 pediatric patients with a median age of 6.22 (0.29, 18.00) years. Fifty‐eight patients (36.25%) were diagnosed with sarcoma. AEs potentially related to PEG‐rhG‐CSF included bone pain ( n = 32), fatigue ( n = 21), pain at the injection site ( n = 21), and myalgia ( n = 20). The rates of grade 3/4 neutropenia and FN during treatment were 57.28% and 29.45%, respectively. Conclusion PEG‐rhG‐CSF is well tolerated and effective in pediatric patients with solid tumors or NHL. These findings should be substantiated with further trials. Clinical Trial Registration ClinicalTrials.gov identifier: NCT04547829.