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Neuroimmune disorders of the central nervous system in children in the molecular era

2018; Nature Portfolio; Volume: 14; Issue: 7 Linguagem: Inglês

10.1038/s41582-018-0024-9

1759-4766

Elizabeth Wells, Yael Hacohen, Amy Waldman, Jan Mendelt Tillema, Ariane Soldatos, Beau M. Ances, Susanne M. Benseler, Bibi Bielekova, Russell C. Dale, Josep Dalmau, William D. Gaillard, Mark Gorman, Benjamin Greenberg, Ann Hyslop, Carlos A. Pardo, Robert C. Tasker, E. Ann Yeh, Amit Bar‐Or, Sean J. Pittock, Adeline Vanderver, Brenda Banwell, Jessica L. Carpenter, Irene Cortese, Nathan C. Dean, Racquel Farias-Moeller, William Gallentine, Carol Glaser, Raphaela Goldbach‐Mansky, Ilana Kahn, Bennett Lavenstein, W. McClintock, William McDow, Jennifer Murphy, Avindra Nath, Roger J. Packer, Tova Ronis, David Schleyer, Stephanie Schleyer, Peter Shibuya, Ursula Utz, Gilbert Vézina, David Wessel,

interferon and immune responses

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. In this Review, the authors discuss all aspects of immune-mediated disorders of the CNS in children, from the clinical features and treatment to pathological mechanisms and biomarkers, and outline priorities for collaborative research to develop precision medicine for these disorders.