Efficacy, safety, and cost-effectiveness of pegylated PEG-rhg-CSF in pediatric patients receiving high-intensity chemotherapy: results from a phase II study

• Published in: Frontiers in pharmacology Vol. 15; p. 1419369
• Main Authors: Huang, Junting, Zhu, Jia, Jiang, Lian, Xu, Jiaqian, Lin, Xiheng, Chang, Jian, Zhang, Xiaohong, Lu, Suying, Sun, Feifei, Wang, Juan, Que, Yi, Ye, Zhonglv, Yang, Lihua, Yuan, Xiuli, Cai, Weisong, Tian, Chuan, Wu, Yanpeng, He, Xiangling, Tang, Yan-Lai, Zhang, Yizhuo
• Format: Journal Article
• Language: English
• Published: Switzerland Frontiers Media S.A 17.07.2024
• Subjects: high-intensity chemotherapy; neutropenia; pediatric cancer patients; PEG rhG-CSF; Pharmacology; phase ii study; pediatric cancer patients; phase ii study; neutropenia; PEG rhG-CSF; high-intensity chemotherapy
• ISSN: 1663-9812; 1663-9812
• DOI: 10.3389/fphar.2024.1419369

High-intensity chemotherapy can cause life-threatening complications in pediatric patients. Therefore, this study investigated safety and efficacy of long-acting pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF; Jinyouli ) in children undergoing high-intensity chemotherapy. Treatment-naive patients received post-chemotherapy PEG-rhG-CSF as primary prophylaxis for two cycles. The primary endpoints were drug-related adverse events (AEs) and bone pain scores. Secondary endpoints included grade 3-4 neutropenia, duration of neutropenia recovery, absolute neutrophil count changes, febrile neutropenia (FN), reduced chemotherapy intensity, antibiotic usage, and AE severity. The cost-effectiveness of PEG-rhG-CSF was compared with that of rhG-CSF (Ruibai ). Here, 307 and 288 patients underwent one and two PEG-rhG-CSF cycles, respectively. Ninety-one patients experienced drug-related AEs, primarily bone pain (12.7%). Moreover, Grade 3-4 neutropenia and FN were observed. Median FN durations were 3.0 days in both cycles. No drug-related delays were observed during chemotherapy. One patient experienced grade 4 neutropenia-induced reduction in chemotherapy intensity during cycle 2. In total, 138 patients received antibiotics. PEG-rhG-CSF exhibited superior cost-effectiveness compared to rhG-CSF. Our findings indicate that PEG-rhG-CSF is safe, efficient, and cost-effective in pediatric patients undergoing high-intensity chemotherapy, providing preliminary evidence warranting further randomized controlled trials.