Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China

BackgroundProgrammed death receptor 1 (PD-1) inhibitors have shown durable response and mild adverse events in adult malignancies. However, study on PD-1 inhibitors in pediatric patients remains limited, and a direct comparison of distinct PD-1 inhibitors in pediatric tumors is lacking.MethodsWe con...

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Main Authors: Lei Mao, Mengzhen Li, Linnan Wu, Juan Wang, Yi Que, Feifei Sun, Junting Huang, Suying Lu, Zijun Zhen, Jia Zhu, Mengjia Song, Yizhuo Zhang
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-06-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1608844/full
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author Lei Mao
Mengzhen Li
Linnan Wu
Juan Wang
Yi Que
Feifei Sun
Junting Huang
Suying Lu
Zijun Zhen
Jia Zhu
Mengjia Song
Yizhuo Zhang
author_facet Lei Mao
Mengzhen Li
Linnan Wu
Juan Wang
Yi Que
Feifei Sun
Junting Huang
Suying Lu
Zijun Zhen
Jia Zhu
Mengjia Song
Yizhuo Zhang
author_sort Lei Mao
collection DOAJ
description BackgroundProgrammed death receptor 1 (PD-1) inhibitors have shown durable response and mild adverse events in adult malignancies. However, study on PD-1 inhibitors in pediatric patients remains limited, and a direct comparison of distinct PD-1 inhibitors in pediatric tumors is lacking.MethodsWe conducted a retrospective analysis of 75 pediatric patients with advanced or recurrent malignancies treated with either Sintilimab-based (n=53) or Pembrolizumab-based (n=22) regimens. The primary endpoints included treatment-related adverse events (TRAEs) and objective response rate (ORR), and the second endpoints included progression-free survival (PFS) and overall survival (OS).ResultsThe incidence of hypothyroidism, hyperthyroidism, pneumonia, increased ALT/AST, gastroenteritis, and rash following immune checkpoint inhibitor therapy showed no significant differences between the Sintilimab group and the Pembrolizumab group (all P>0.05). Cardiovascular Adverse Events (CVAEs) occurred in 26.0% (15/53) of Sintilimab-treated patients versus 40.0% (8/20) of Pembrolizumab-treated patients (P=0.26). In the lymphoma cohort (n=13), 88.9% of Sintilimab-treated patients and 75.0% of Pembrolizumab-treated patients achieved complete response (CR) or partial response (PR) (P=0.54). The median PFS and OS were not reached in either group. In the non-lymphoma cohort (n=53), 40.5% of Sintilimab-treated patients and 25.0% of Pembrolizumab-treated patients achieved CR or PR (P=0.18). Among 39 patients who had received ≤ 2 prior treatment lines, the PFS and OS showed no significant differences between the Sintilimab (n=30) and Pembrolizumab (n=9) groups (P=0.28 and P=0.09, respectively). Similarly, among 14 patients who had received>2 prior treatment lines, no significant differences in PFS and OS were observed between the Sintilimab(n=7) and Pembrolizumab(n=7) groups (P=0.33 and P=0.15, respectively).ConclusionsSintilimab demonstrated favorable tolerability and efficacy in pediatric patients with malignancies, with a safety and efficacy profile comparable to Pembrolizumab. For pediatric patients with advanced or recurrent malignancies receiving immune checkpoint inhibitor therapy, long-term monitoring of thyroid and cardiac function is recommended.
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spelling doaj-art-b4d91f676fa14e428fdd5d45093dbe7d2025-08-20T03:26:20ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-06-011610.3389/fimmu.2025.16088441608844Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in ChinaLei MaoMengzhen LiLinnan WuJuan WangYi QueFeifei SunJunting HuangSuying LuZijun ZhenJia ZhuMengjia SongYizhuo ZhangBackgroundProgrammed death receptor 1 (PD-1) inhibitors have shown durable response and mild adverse events in adult malignancies. However, study on PD-1 inhibitors in pediatric patients remains limited, and a direct comparison of distinct PD-1 inhibitors in pediatric tumors is lacking.MethodsWe conducted a retrospective analysis of 75 pediatric patients with advanced or recurrent malignancies treated with either Sintilimab-based (n=53) or Pembrolizumab-based (n=22) regimens. The primary endpoints included treatment-related adverse events (TRAEs) and objective response rate (ORR), and the second endpoints included progression-free survival (PFS) and overall survival (OS).ResultsThe incidence of hypothyroidism, hyperthyroidism, pneumonia, increased ALT/AST, gastroenteritis, and rash following immune checkpoint inhibitor therapy showed no significant differences between the Sintilimab group and the Pembrolizumab group (all P>0.05). Cardiovascular Adverse Events (CVAEs) occurred in 26.0% (15/53) of Sintilimab-treated patients versus 40.0% (8/20) of Pembrolizumab-treated patients (P=0.26). In the lymphoma cohort (n=13), 88.9% of Sintilimab-treated patients and 75.0% of Pembrolizumab-treated patients achieved complete response (CR) or partial response (PR) (P=0.54). The median PFS and OS were not reached in either group. In the non-lymphoma cohort (n=53), 40.5% of Sintilimab-treated patients and 25.0% of Pembrolizumab-treated patients achieved CR or PR (P=0.18). Among 39 patients who had received ≤ 2 prior treatment lines, the PFS and OS showed no significant differences between the Sintilimab (n=30) and Pembrolizumab (n=9) groups (P=0.28 and P=0.09, respectively). Similarly, among 14 patients who had received>2 prior treatment lines, no significant differences in PFS and OS were observed between the Sintilimab(n=7) and Pembrolizumab(n=7) groups (P=0.33 and P=0.15, respectively).ConclusionsSintilimab demonstrated favorable tolerability and efficacy in pediatric patients with malignancies, with a safety and efficacy profile comparable to Pembrolizumab. For pediatric patients with advanced or recurrent malignancies receiving immune checkpoint inhibitor therapy, long-term monitoring of thyroid and cardiac function is recommended.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1608844/fullpediatric malignanciesPD-1 inhibitorssintilimabpembrolizumabsafetyefficacy
spellingShingle Lei Mao
Mengzhen Li
Linnan Wu
Juan Wang
Yi Que
Feifei Sun
Junting Huang
Suying Lu
Zijun Zhen
Jia Zhu
Mengjia Song
Yizhuo Zhang
Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
Frontiers in Immunology
pediatric malignancies
PD-1 inhibitors
sintilimab
pembrolizumab
safety
efficacy
title Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
title_full Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
title_fullStr Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
title_full_unstemmed Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
title_short Safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies: a real-world study in China
title_sort safety and efficacy of sintilimab versus pembrolizumab in the treatment of advanced or recurrent pediatric malignancies a real world study in china
topic pediatric malignancies
PD-1 inhibitors
sintilimab
pembrolizumab
safety
efficacy
url https://www.frontiersin.org/articles/10.3389/fimmu.2025.1608844/full
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