PMID- 37359533 OWN - NLM STAT- MEDLINE DCOM- 20230628 LR - 20230701 IS - 1664-3224 (Electronic) IS - 1664-3224 (Linking) VI - 14 DP - 2023 TI - Efficacy and safety of programmed cell death receptor 1 inhibition-based regimens in patients with pediatric malignancies: the real-world study in China. PG - 1182751 LID - 10.3389/fimmu.2023.1182751 [doi] LID - 1182751 AB - BACKGROUND: Programmed 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. METHODS: We 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. RESULTS: A 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. CONCLUSIONS: This 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. CI - Copyright (c) 2023 Hong, Song, Lan, Wang, Lu, Zhang, Zhu, Sun, Huang, Liu, Xu, Wu, Guo, Cai, Zhen, Que and Zhang. FAU - Hong, Ye AU - Hong Y AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Song, Mengjia AU - Song M AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Lan, Yingxia AU - Lan Y AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Wang, Juan AU - Wang J AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Lu, Suying AU - Lu S AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Zhang, Yu AU - Zhang Y AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Zhu, Jia AU - Zhu J AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Sun, Feifei AU - Sun F AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Huang, Junting AU - Huang J AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Liu, Juan AU - Liu J AD - Department of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, China. FAU - Xu, Jiaqian AU - Xu J AD - Department of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, China. FAU - Wu, Yanpeng AU - Wu Y AD - Department of Pediatric, The Fifth Affiliated Hospital, Guangzhou Medical University, Guangzhou, Guang Dong, China. FAU - Guo, Haixia AU - Guo H AD - Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Cai, Ruiqing AU - Cai R AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Zhen, Zijun AU - Zhen Z AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Que, Yi AU - Que Y AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Zhang, Yizhuo AU - Zhang Y AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20230609 PL - Switzerland TA - Front Immunol JT - Frontiers in immunology JID - 101560960 RN - 0 (Immune Checkpoint Inhibitors) RN - 0 (Programmed Cell Death 1 Receptor) RN - 0 (Vascular Endothelial Growth Factor A) SB - IM MH - Adult MH - Humans MH - Child MH - Retrospective Studies MH - Immune Checkpoint Inhibitors/therapeutic use MH - Programmed Cell Death 1 Receptor MH - Vascular Endothelial Growth Factor A MH - *Carcinoma, Non-Small-Cell Lung/drug therapy MH - *Lung Neoplasms/drug therapy MH - Apoptosis PMC - PMC10288191 OTO - NOTNLM OT - PD-1 inhibitor-based treatment OT - PD-1 inhibitors OT - efficacy OT - pediatric malignancies OT - safety COIS- The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.- EDAT- 2023/06/26 19:07 MHDA- 2023/06/28 06:42 PMCR- 2023/01/01 CRDT- 2023/06/26 12:25 PHST- 2023/03/09 00:00 [received] PHST- 2023/05/29 00:00 [accepted] PHST- 2023/06/28 06:42 [medline] PHST- 2023/06/26 19:07 [pubmed] PHST- 2023/06/26 12:25 [entrez] PHST- 2023/01/01 00:00 [pmc-release] AID - 10.3389/fimmu.2023.1182751 [doi] PST - epublish SO - Front Immunol. 2023 Jun 9;14:1182751. doi: 10.3389/fimmu.2023.1182751. eCollection 2023.