AUTHOR=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 

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

JOURNAL=Frontiers in Pharmacology

VOLUME=Volume 15 - 2024

YEAR=2024

URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2024.1419369

DOI=10.3389/fphar.2024.1419369

ISSN=1663-9812

ABSTRACT=Background: 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.Methods: 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 ® ).Results: 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 drugrelated delays were observed during chemotherapy. One patient experienced grade 4 neutropeniainduced 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.* Duration of neutropenia recovery data were not available for one patient in cycle 1 and for 32 patients in cycle 2. ANC, absolute neutrophil count; FN, febrile neutropenia.