Diagnosis and Management of Opsoclonus-Myoclonus-Ataxia Syndrome in Children: An International Perspective

Thomas Rossor; E Ann Yeh; Yasmin Khakoo; Paola Angelini; Cheryl Hemingway; Sarosh R Irani; Gudrun Schleiermacher; Paramala Santosh; Tim Lotze; Russell C Dale; Kumaran Deiva; Barbara Hero; Andrea Klein; Pedro de Alarcon; Mark P Gorman; Wendy G Mitchell; Ming Lim; OMS Study Group

doi:10.1212/NXI.0000000000001153

Diagnosis and Management of Opsoclonus-Myoclonus-Ataxia Syndrome in Children: An International Perspective

Neurol Neuroimmunol Neuroinflamm. 2022 Mar 8;9(3):e1153. doi: 10.1212/NXI.0000000000001153. Print 2022 May.

Authors

Thomas Rossor¹, E Ann Yeh¹, Yasmin Khakoo¹, Paola Angelini¹, Cheryl Hemingway¹, Sarosh R Irani¹, Gudrun Schleiermacher¹, Paramala Santosh¹, Tim Lotze¹, Russell C Dale¹, Kumaran Deiva¹, Barbara Hero¹, Andrea Klein¹, Pedro de Alarcon¹, Mark P Gorman¹, Wendy G Mitchell¹, Ming Lim²; OMS Study Group

Affiliations

¹ From the Children's Neurosciences (T.R., M.L.), Evelina London Children's Hospital at Guy's and St Thomas' NHS Foundation Trust, King's Health Partners Academic Health Science Centre; Department Women and Children's Health (T.R., M.L.), School of Life Course Sciences (SoLCS), King's College London, UK; Division of Neurology (E.A.Y.), Department of Pediatrics, Neurosciences and Mental Health (RI), The Hospital for Sick Children; Faculty of Medicine (E.A.Y.), The University of Toronto, Ontario, Canada; Department of Pediatrics (Y.K.) and Department of Neurology (Y.K.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Pediatrics (Y.K.), Weill Medical College of Cornell University, New York; Children and Young People's Unit (Paola Angelini), The Royal Marsden, Downs Road, Sutton, Surrey; UCL Great Ormond Street Institute of Child Health (C.H.), Department of Neurology, Great Ormond Street Hospital for Children, London; Oxford Autoimmune Neurology Group (S.R.I.), Nuffield Department of Clinical Neurosciences, University of Oxford; Department of Neurology (S.R.I.), Oxford University Hospitals NHS Foundation Trust, UK; SiRIC RTOP (G.S.), Translational Research Department, PSL Research University, Institut Curie Research Center; INSERM U830 (G.S.), PSL Research University, Institut Curie Research Center; SIREDO Center: Care (G.S.), Innovation and Research for Children, Adolescents and Young Adults with Cancer, Institut Curie, Paris, France; Department of Child and Adolescent Psychiatry (P.S.), King's College London; Centre for Interventional Paediatric Psychopharmacology and Rare Diseases (CIPPRD) Research Team (P.S.), South London and Maudsley NHS Foundation Trust, London, UK; Baylor College of Medicine (T.L.), Texas Children's Hospital, Houston; Kids Neuroscience Centre (R.C.D.), The Children's Hospital at Westmead, Westmead, NSW, Australia; TY Nelson Department of Neurology and Neurosurgery (R.C.D.), The Children's Hospital at Westmead; The Children's Hospital at Westmead Clinical School (R.C.D.), Faculty of Medicine, University of Sydney, NSW, Australia; Pediatric Neurology Department (K.D.), Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Saclay, Bicêtre Hospital; National Referral Center for Rare Inflammatory and Auto-Immune Brain and Spinal Diseases (K.D.); Inserm UMR 1184 (K.D.), Immunology of Viral Infections and Autoimmune Diseases, CEA, IDMIT, Le Kremlin Bicêtre, France; Department of Pediatric Hematology and Oncology (B.H.), University Children's Hospital, Koln; Division of Child Neurology (A.K.), University Children's Hospital Bern Inselspital, University of Bern; Department of Pediatric Neurology (A.K.), University Children's Hospital Basel, Switzerland; Department of Pediatrics (Pedro de Alarcon), University of Illinois College of Medicine at Peoria, Peoria IL; Department of Neurology (M.P.G.), Boston Children's Hospital, Harvard Medical School, MA; Division of Neurology (W.G.M.), Department of Pediatrics, Children's Hospital Los Angeles; and Department of Neurology (W.G.M.), Keck School of Medicine at the University of Southern California, Los Angeles.
² From the Children's Neurosciences (T.R., M.L.), Evelina London Children's Hospital at Guy's and St Thomas' NHS Foundation Trust, King's Health Partners Academic Health Science Centre; Department Women and Children's Health (T.R., M.L.), School of Life Course Sciences (SoLCS), King's College London, UK; Division of Neurology (E.A.Y.), Department of Pediatrics, Neurosciences and Mental Health (RI), The Hospital for Sick Children; Faculty of Medicine (E.A.Y.), The University of Toronto, Ontario, Canada; Department of Pediatrics (Y.K.) and Department of Neurology (Y.K.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Pediatrics (Y.K.), Weill Medical College of Cornell University, New York; Children and Young People's Unit (Paola Angelini), The Royal Marsden, Downs Road, Sutton, Surrey; UCL Great Ormond Street Institute of Child Health (C.H.), Department of Neurology, Great Ormond Street Hospital for Children, London; Oxford Autoimmune Neurology Group (S.R.I.), Nuffield Department of Clinical Neurosciences, University of Oxford; Department of Neurology (S.R.I.), Oxford University Hospitals NHS Foundation Trust, UK; SiRIC RTOP (G.S.), Translational Research Department, PSL Research University, Institut Curie Research Center; INSERM U830 (G.S.), PSL Research University, Institut Curie Research Center; SIREDO Center: Care (G.S.), Innovation and Research for Children, Adolescents and Young Adults with Cancer, Institut Curie, Paris, France; Department of Child and Adolescent Psychiatry (P.S.), King's College London; Centre for Interventional Paediatric Psychopharmacology and Rare Diseases (CIPPRD) Research Team (P.S.), South London and Maudsley NHS Foundation Trust, London, UK; Baylor College of Medicine (T.L.), Texas Children's Hospital, Houston; Kids Neuroscience Centre (R.C.D.), The Children's Hospital at Westmead, Westmead, NSW, Australia; TY Nelson Department of Neurology and Neurosurgery (R.C.D.), The Children's Hospital at Westmead; The Children's Hospital at Westmead Clinical School (R.C.D.), Faculty of Medicine, University of Sydney, NSW, Australia; Pediatric Neurology Department (K.D.), Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Saclay, Bicêtre Hospital; National Referral Center for Rare Inflammatory and Auto-Immune Brain and Spinal Diseases (K.D.); Inserm UMR 1184 (K.D.), Immunology of Viral Infections and Autoimmune Diseases, CEA, IDMIT, Le Kremlin Bicêtre, France; Department of Pediatric Hematology and Oncology (B.H.), University Children's Hospital, Koln; Division of Child Neurology (A.K.), University Children's Hospital Bern Inselspital, University of Bern; Department of Pediatric Neurology (A.K.), University Children's Hospital Basel, Switzerland; Department of Pediatrics (Pedro de Alarcon), University of Illinois College of Medicine at Peoria, Peoria IL; Department of Neurology (M.P.G.), Boston Children's Hospital, Harvard Medical School, MA; Division of Neurology (W.G.M.), Department of Pediatrics, Children's Hospital Los Angeles; and Department of Neurology (W.G.M.), Keck School of Medicine at the University of Southern California, Los Angeles. ming.lim@gstt.nhs.uk.

Abstract

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.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Ataxia / complications
Child
Disease Progression
Humans
Internationality
Neuroblastoma* / diagnosis
Neuroblastoma* / drug therapy
Ocular Motility Disorders* / complications
Opsoclonus-Myoclonus Syndrome* / complications
Opsoclonus-Myoclonus Syndrome* / diagnosis
Opsoclonus-Myoclonus Syndrome* / therapy

Abstract

Publication types

MeSH terms

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