Center for Neuroscience and Behavioral Medicine, Children's National Health System, Washington, DC, USA.
Department of Neuroinflammation, Queen Square MS Centre, UCL Institute of Neurology, London, UK.
Nat Rev Neurol. 2018 Jul;14(7):433-445. doi: 10.1038/s41582-018-0024-9.
Immune-mediated disorders of the CNS in children are a complex group of demyelinating, inflammatory, parainfectious and postinfectious disorders with heterogeneous pathobiological mechanisms and clinical manifestations, often associated with fundamental derangement in immune regulation. In this Review, we aim to provide an update on our knowledge of neuroimmune disorders and highlight areas of research that are priorities for improving clinical management. We outline the clinical features of neuroimmune disorders, the current approaches to their treatment and new approaches in development. We then consider the pathological features, including biomarkers, pathological mechanisms and genetics, and discuss the value of immune assays in clinical investigation and basic research. On the basis of current knowledge and techniques, we propose four research priorities: rigorous and consistent collection of core clinical data, cooperative investigation of treatments, development of biological assays and genetic studies. These priorities should help us to achieve the shared goal of precision medicine for neuroimmune disorders. However, multicentre research and the creation of clinical consortia for these rare disorders will be necessary, and we hope that this Review serves as a call to action that is timely given current exciting advances in neuroimmune therapeutics.
儿童中枢神经系统免疫介导疾病是一组复杂的脱髓鞘、炎症、副感染和感染后疾病,具有不同的病理生物学机制和临床表现,常与免疫调节的根本紊乱有关。在这篇综述中,我们旨在提供对神经免疫性疾病的最新认识,并强调优先研究的领域,以改善临床管理。我们概述了神经免疫性疾病的临床特征、目前的治疗方法和新的开发方法。然后,我们考虑了病理特征,包括生物标志物、病理机制和遗传学,并讨论了免疫检测在临床研究和基础研究中的价值。基于目前的知识和技术,我们提出了四项研究重点:严格和一致地收集核心临床数据、合作研究治疗方法、开发生物检测方法和进行遗传研究。这些重点应该有助于我们实现神经免疫性疾病精准医学的共同目标。然而,对于这些罕见疾病,需要进行多中心研究和建立临床联盟,我们希望这篇综述能够及时呼吁采取行动,因为神经免疫治疗目前正在取得令人兴奋的进展。