TY - JOUR
T1 - Neuroimmune disorders of the central nervous system in children in the molecular era
AU - Wells, Elizabeth
AU - Hacohen, Yael
AU - Waldman, Amy
AU - Tillema, Jan M.
AU - Soldatos, Ariane
AU - Ances, Beau
AU - Benseler, Susanne
AU - Bielekova, Bibi
AU - Dale, Russel C.
AU - Dalmau, Josep
AU - Gaillard, William
AU - Gorman, Mark
AU - Greenberg, Benjamin
AU - Hyslop, Ann
AU - Pardo, Carlos A.
AU - Tasker, Robert C.
AU - Yeh, E. Ann
AU - Bar-Or, Amit
AU - Pittock, Sean
AU - Vanderver, Adeline
AU - Banwell, Brenda
N1 - Funding Information:
This Review was derived from a meeting of clinicians, scientists and patient advocacy groups that was held in Washington DC in June 2014 and was funded by the Ikaria Fund at Children’s National Health System. A.W. has received research support from the Children’s Hospital of Philadelphia (Foerderer Award), Elise’s Corner (the Akron Community Foundation), the Lynn Saligman League, the US NIH (K23NS069806 and R01NS071463), the National Multiple Sclerosis Society and the Calliope Joy Foundation. B.A. is funded by the US NIH (R01 NR012907, R01 NR014449, R01 NR014449 and R01 NR015738). R.C.D. has received research funding from MS Research Australia, the Australian National Health and Medical Research Council, the Petre Foundation, the Tourette Syndrome Association and the University of Sydney. W.G. receives grant support from the American Epilepsy Society, Citizens United for Research in Epilepsy, the Epilepsy Foundation, the Infantile Epilepsy Research Foundation, the US National Science Foundation, the US NIH and the Patient-Centered Outcomes Research Institute. B.G. has grant support from the Guthy-Jackson Charitable Foundation, the US NIH and the Patient-Centered Outcomes Research Institute. R.C.T. has received funding from the US NIH (R21-NS084264 and U01-NS081041). E.A.Y. has received funding from the Canadian Institute for Health Information, the Canadian Institutes for Health Research, the Mario Batali Foundation, the Multiple Sclerosis Society of Canada, the National Multiple Sclerosis Society, the Patient-Centered Outcomes Research Institute, the Rare Disease Foundation, the SickKids Foundation and the SickKids Innovation Fund. She has received travel support and/or honoraria from the Paediatric Association of Jamaica, the Saudi Arabian Child Neurology Association, the Guthy-Jackson Charitable Foundation and the Nicholas Foundation. S.P. has received support from the Guthy-Jackson Charitable Foundation and the US NIH (R01 NS065829-01) for neuromyelitis optica research.
Publisher Copyright:
© 2018 Macmillan Publishers Ltd., part of Springer Nature.
PY - 2018/7/1
Y1 - 2018/7/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85048707895&partnerID=8YFLogxK
U2 - 10.1038/s41582-018-0024-9
DO - 10.1038/s41582-018-0024-9
M3 - Review article
C2 - 29925924
AN - SCOPUS:85048707895
SN - 1759-4758
VL - 14
SP - 433
EP - 445
JO - Nature Reviews Neurology
JF - Nature Reviews Neurology
IS - 7
ER -