脑肿瘤是儿童中最常见的实体瘤，也是癌症相关死亡的主要原因。过去几年来，对中枢神经系统肿瘤患儿的诊断和治疗取得了改进。然而，对许多高级别肿瘤患者而言，总体预后仍然较差。较低的生存率部分归因于缺乏有效的治疗方法。在成人身上，免疫检查点抑制剂（ICIs）的问世和成功引发了对这些治疗方法在儿童患者中单独应用或与其他药物治疗相结合的研究兴趣。然而，要实现改善的临床预后，建立和选择相关而强有力的临床前儿童高级别脑肿瘤模型至关重要。在这里，我们回顾了影响模型选择的信息，因为我们开始了一项国际合作研究，测试ICIs与表观遗传修饰剂的联合应用，以增强适应性免疫力，用于治疗儿童脑肿瘤。我们还分享了我们所面临的挑战和潜在解决方案。© 作者们 2023. 版权归牛津大学出版社代表神经肿瘤学会所有。保留所有权利。如需许可，请发送电子邮件至：journals.permissions@oup.com。

Brain tumors are the most common solid tumor in children and the leading cause of cancer-related deaths. Over the last few years, improvements have been made in the diagnosis and treatment of children with Central Nervous System tumors. Unfortunately, for many patients with high-grade tumors, the overall prognosis remains poor. Lower survival rates are partly attributed to the lack of efficacious therapies. The advent and success of immune checkpoint inhibitors (ICIs) in adults have sparked interest in investigating the utility of these therapies alone or in combination with other drug treatments in pediatric patients. However, to achieve improved clinical outcomes, the establishment and selection of relevant and robust preclinical pediatric high-grade brain tumor models is imperative. Here we review the information that influenced our model selection as we embarked on an international collaborative study to test ICIs in combination with epigenetic modifying agents to enhance adaptive immunity to treat pediatric brain tumors. We also share challenges that we faced and potential solutions.© The Author(s) 2023. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.