Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China
BackgroundProgrammed death receptor 1 (PD-1) inhibition has shown durable response and mild adverse events (AEs) in adult malignancies. However, data on the clinical activity of PD-1 inhibition in pediatric patients are lacking. We comprehensively assessed the efficacy and safety of PD-1 inhibitor-b...
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Frontiers Media S.A.
2023-06-01
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| Series: | Frontiers in Immunology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fimmu.2023.1182751/full |
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| author | Ye Hong Ye Hong Mengjia Song Mengjia Song Yingxia Lan Yingxia Lan Juan Wang Juan Wang Suying Lu Suying Lu Yu Zhang Yu Zhang Jia Zhu Jia Zhu Feifei Sun Feifei Sun Junting Huang Junting Huang Juan Liu Jiaqian Xu Yanpeng Wu Haixia Guo Ruiqing Cai Ruiqing Cai Zijun Zhen Zijun Zhen Yi Que Yi Que Yizhuo Zhang Yizhuo Zhang |
| author_facet | Ye Hong Ye Hong Mengjia Song Mengjia Song Yingxia Lan Yingxia Lan Juan Wang Juan Wang Suying Lu Suying Lu Yu Zhang Yu Zhang Jia Zhu Jia Zhu Feifei Sun Feifei Sun Junting Huang Junting Huang Juan Liu Jiaqian Xu Yanpeng Wu Haixia Guo Ruiqing Cai Ruiqing Cai Zijun Zhen Zijun Zhen Yi Que Yi Que Yizhuo Zhang Yizhuo Zhang |
| author_sort | Ye Hong |
| collection | DOAJ |
| description | BackgroundProgrammed death receptor 1 (PD-1) inhibition has shown durable response and mild adverse events (AEs) in adult malignancies. However, data on the clinical activity of PD-1 inhibition in pediatric patients are lacking. We comprehensively assessed the efficacy and safety of PD-1 inhibitor-based regimens for pediatric malignancies.MethodsWe conducted a real-world, multi-institutional, retrospective analysis of pediatric malignancies treated with PD-1 inhibitor-based regimens. The primary endpoints were objective response rate (ORR) and progression-free survival (PFS). The secondary endpoints included disease control rate (DCR), duration of response (DOR), and AEs. The Kaplan–Meier method was used to calculate PFS and DOR. The National Cancer Institute Common Toxicity Criteria for AEs (version 5.0) were used to grade toxicity.ResultsA total of 93 and 109 patients were evaluated for efficacy and safety, respectively. For all efficacy-evaluable patients, PD-1 inhibitor monotherapy, combined chemotherapy, combined histone deacetylase inhibitor, and combined vascular endothelial growth factor receptor tyrosine kinase inhibitor cohorts, the ORR and DCR were 53.76%/81.72%, 56.67%/83.33%, 54.00%/80.00%, 100.00%/100.00%, and 12.50%/75.00%, respectively; the median PFS and DOR were 17.6/31.2 months, not achieved/not achieved, 14.9/31.2 months, 17.6/14.9 months, and 3.7/1.8 months, respectively; the incidence rate of AEs were 83.49%, 55.26%, 100.00%, 80.00%, and 100.00%, respectively. One patient in the PD-1 inhibitor-combined chemotherapy cohort discontinued treatment due to diabetic ketoacidosis.ConclusionsThis largest retrospective analysis demonstrate that PD-1 inhibitor-based regimens are potentially effective and tolerable in pediatric malignancies. Our findings provide references for future clinical trials and practice of PD-1 inhibitors in pediatric cancer patients. |
| first_indexed | 2024-03-13T06:35:41Z |
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| id | doaj.art-14338471116648688dafaf8de3f04e25 |
| institution | Directory Open Access Journal |
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| language | English |
| last_indexed | 2024-03-13T06:35:41Z |
| publishDate | 2023-06-01 |
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| spelling | doaj.art-14338471116648688dafaf8de3f04e252023-06-09T04:44:59ZengFrontiers Media S.A.Frontiers in Immunology1664-32242023-06-011410.3389/fimmu.2023.11827511182751Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in ChinaYe Hong0Ye Hong1Mengjia Song2Mengjia Song3Yingxia Lan4Yingxia Lan5Juan Wang6Juan Wang7Suying Lu8Suying Lu9Yu Zhang10Yu Zhang11Jia Zhu12Jia Zhu13Feifei Sun14Feifei Sun15Junting Huang16Junting Huang17Juan Liu18Jiaqian Xu19Yanpeng Wu20Haixia Guo21Ruiqing Cai22Ruiqing Cai23Zijun Zhen24Zijun Zhen25Yi Que26Yi Que27Yizhuo Zhang28Yizhuo Zhang29State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, ChinaDepartment of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, ChinaDepartment of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, ChinaDepartment of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaState Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaBackgroundProgrammed death receptor 1 (PD-1) inhibition has shown durable response and mild adverse events (AEs) in adult malignancies. However, data on the clinical activity of PD-1 inhibition in pediatric patients are lacking. We comprehensively assessed the efficacy and safety of PD-1 inhibitor-based regimens for pediatric malignancies.MethodsWe conducted a real-world, multi-institutional, retrospective analysis of pediatric malignancies treated with PD-1 inhibitor-based regimens. The primary endpoints were objective response rate (ORR) and progression-free survival (PFS). The secondary endpoints included disease control rate (DCR), duration of response (DOR), and AEs. The Kaplan–Meier method was used to calculate PFS and DOR. The National Cancer Institute Common Toxicity Criteria for AEs (version 5.0) were used to grade toxicity.ResultsA total of 93 and 109 patients were evaluated for efficacy and safety, respectively. For all efficacy-evaluable patients, PD-1 inhibitor monotherapy, combined chemotherapy, combined histone deacetylase inhibitor, and combined vascular endothelial growth factor receptor tyrosine kinase inhibitor cohorts, the ORR and DCR were 53.76%/81.72%, 56.67%/83.33%, 54.00%/80.00%, 100.00%/100.00%, and 12.50%/75.00%, respectively; the median PFS and DOR were 17.6/31.2 months, not achieved/not achieved, 14.9/31.2 months, 17.6/14.9 months, and 3.7/1.8 months, respectively; the incidence rate of AEs were 83.49%, 55.26%, 100.00%, 80.00%, and 100.00%, respectively. One patient in the PD-1 inhibitor-combined chemotherapy cohort discontinued treatment due to diabetic ketoacidosis.ConclusionsThis largest retrospective analysis demonstrate that PD-1 inhibitor-based regimens are potentially effective and tolerable in pediatric malignancies. Our findings provide references for future clinical trials and practice of PD-1 inhibitors in pediatric cancer patients.https://www.frontiersin.org/articles/10.3389/fimmu.2023.1182751/fullpediatric malignanciesPD-1 inhibitorsPD-1 inhibitor-based treatmentsafetyefficacy |
| spellingShingle | Ye Hong Ye Hong Mengjia Song Mengjia Song Yingxia Lan Yingxia Lan Juan Wang Juan Wang Suying Lu Suying Lu Yu Zhang Yu Zhang Jia Zhu Jia Zhu Feifei Sun Feifei Sun Junting Huang Junting Huang Juan Liu Jiaqian Xu Yanpeng Wu Haixia Guo Ruiqing Cai Ruiqing Cai Zijun Zhen Zijun Zhen Yi Que Yi Que Yizhuo Zhang Yizhuo Zhang Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China Frontiers in Immunology pediatric malignancies PD-1 inhibitors PD-1 inhibitor-based treatment safety efficacy |
| title | Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China |
| title_full | Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China |
| title_fullStr | Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China |
| title_full_unstemmed | Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China |
| title_short | Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China |
| title_sort | efficacy and safety of programmed cell death receptor 1 inhibition based regimens in patients with pediatric malignancies the real world study in china |
| topic | pediatric malignancies PD-1 inhibitors PD-1 inhibitor-based treatment safety efficacy |
| url | https://www.frontiersin.org/articles/10.3389/fimmu.2023.1182751/full |
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