RT Journal Article
SR Electronic
T1 Diagnosis and Management of Opsoclonus-Myoclonus-Ataxia Syndrome in Children
JF Neurology - Neuroimmunology Neuroinflammation
JO Neurol Neuroimmunol Neuroinflamm
FD Lippincott Williams & Wilkins
SP e1153
DO 10.1212/NXI.0000000000001153
VO 9
IS 3
A1 Thomas Rossor
A1 E. Ann Yeh
A1 Yasmin Khakoo
A1 Paola Angelini
A1 Cheryl Hemingway
A1 Sarosh R. Irani
A1 Gudrun Schleiermacher
A1 Paramala Santosh
A1 Tim Lotze
A1 Russell C. Dale
A1 Kumaran Deiva
A1 Barbara Hero
A1 Andrea Klein
A1 Pedro de Alarcon
A1 Mark P. Gorman
A1 Wendy G. Mitchell
A1 Ming Lim
A1 ,
YR 2022
UL http://nn.neurology.org/content/9/3/e1153.abstract
AB Background and Objectives Opsoclonus-myoclonus-ataxia syndrome (OMAS) is a rare disorder of the nervous system that classically presents with a combination of characteristic eye movement disorder and myoclonus, in addition to ataxia, irritability, and sleep disturbance. There is good evidence that OMAS is an immune-mediated condition that may be paraneoplastic in the context of neuroblastoma. This syndrome may be associated with long-term cognitive impairment, yet it remains unclear how this is influenced by disease course and treatment. Treatment is largely predicated on immune suppression, but there is limited evidence to indicate an optimal regimen.Methods Following an international multiprofessional workshop in 2004, a body of clinicians and scientists comprising the International OMS Study group continued to meet biennially in a joint professionals and family workshop focusing on pediatric OMAS. Seventeen years after publication of the first report, a writing group was convened to provide a clinical update on the definitions and clinical presentation of OMAS, biomarkers and the role of investigations in a child presenting with OMAS, treatment and management strategies including identification and support of long-term sequelae.Results The clinical criteria for diagnosis were reviewed, with a proposed approach to laboratory and radiologic investigation of a child presenting with possible OMAS. The evidence for an upfront vs escalating treatment regimen was reviewed, and a treatment algorithm proposed to recognize both these approaches. Importantly, recommendations on monitoring of immunotherapy response and longer-term follow-up based on an expert consensus are provided.Discussion OMAS is a rare neurologic condition that can be associated with poor cognitive outcomes. This report proposes an approach to investigation and treatment of children presenting with OMAS, based on expert international opinion recognizing the limited data available.ACTH=adrenocorticotropic hormone; ADS=acquired demyelinating syndrome; IVIg=IV immunoglobulin; MIBG=meta-iodo-benzyl-guanidine scan; OMA=opsoclonus-myoclonus-ataxia; OMAS=opsoclonus-myoclonus-ataxia syndrome; OMS=opsoclonus-myoclonus syndrome; OS=overall survival