An International Pediatric-Onset Opsoclonus-Myoclonus Ataxia Syndrome Registry and Clinical Research Network: Development, Progress, and Vision

Opsoclonus-myoclonus ataxia syndrome (OMAS) is a rare neuroimmunological disorder that affects one in 5 million children per year, 1 Pang K.K. de Sousa C. Lang B. Pike M.G. A prospective study of the presentation and management of dancing eye syndrome/opsoclonus-myoclonus syndrome in the United Kingdom. Eur J Paediatr Neurol. 2010; 14: 156-161 Abstract Full Text Full Text PDF PubMed Scopus (89) Google Scholar typically presenting in the second year of life with chaotic eye movements (opsoclonus), muscle jerks (myoclonus), impaired motor coordination (ataxia), and irritability. 2 Hero B. Schleiermacher G. Update on pediatric opsoclonus myoclonus syndrome. Neuropediatrics. 2013; 44: 324-329 Crossref PubMed Scopus (42) Google Scholar Although previous studies suggest that outcomes tend to improve with early intensive immunosuppression, 3 Gorman M.P. Update on diagnosis, treatment, and prognosis in opsoclonus-myoclonus-ataxia syndrome. Curr Opin Pediatr. 2010; 22: 745-750 Crossref PubMed Scopus (112) Google Scholar , 4 Dale R.C. Brilot F. Duffy L.V. et al. Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease. Neurology. 2014; 83: 142-150 Crossref PubMed Scopus (258) Google Scholar , 5 Mitchell W.G. Wooten A.A. O'Neil S.H. Rodriguez J.G. Cruz R.E. Wittern R. Effect of increased immunosuppression on developmental outcome of opsoclonus myoclonus syndrome (OMS). J Child Neurol. 2015; 30: 976-982 Crossref PubMed Scopus (63) Google Scholar a steering committee of pediatric neurologists and oncologists involved with the treatment of OMAS devised plans to organize an expanded research group from 2018 to 2020 and establish an international pediatric-onset opsoclonus-myoclonus ataxia syndrome (POOMAS) registry.