Long-term follow-up of neuroblastoma-associated opsoclonus-myoclonus-ataxia syndrome

Neuropediatrics. 2009 Jun;40(3):103-11. doi: 10.1055/s-0029-1237723. Epub 2009 Dec 17.


Objective: The aim of this study is to describe the long-term neurological, neuropsychological and neuroradiological sequelae and to determine prognostic factors for neurological outcome in children with neuroblastoma-associated opsoclonus-myoclonus-ataxia (OMA) syndrome.

Methods: Data on medical history were collected for the study patients. Examinations with grading of neurological signs, neuropsychological tests and brain magnetic resonance imaging with spectroscopy were performed during a follow-up clinic.

Results: Fourteen subjects entered the study. All had localized neuroblastoma and they were evaluated after a median of 7.8 years. Patients with a chronic/multiphasic neurological course received steroids combined with intravenous immunoglobulins in the majority of cases. 71% presented neurological sequelae and 62% had a full-scale IQ below the normal range. All patients showed at least some deficit in the neuropsychological functions assessed (language, visual-motor integration, memory, attention and motor ability). Long-term deficits were more frequently detected in patients with an interval of more than 2 months between OMA onset and its diagnosis, even if in most comparisons statistical significance was not reached. Cerebellar atrophy, observed in 36% of patients, was not associated with the neurological outcome.

Conclusions: Persisting disability is present in most children with neuroblastoma-associated OMA. However, our results support the role of an early diagnosis of OMA in reducing sequelae and encourage the use of new immunosuppressive therapies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brain Neoplasms / complications*
  • Brain Neoplasms / diagnostic imaging
  • Child
  • Child, Preschool
  • Cognition Disorders / etiology
  • Disease Progression
  • Female
  • Humans
  • Immunoglobulins / administration & dosage
  • Intelligence Tests
  • Longitudinal Studies
  • Male
  • Neuroblastoma / complications*
  • Neuroblastoma / diagnostic imaging
  • Neurologic Examination
  • Neuropsychological Tests
  • Opsoclonus-Myoclonus Syndrome / complications*
  • Opsoclonus-Myoclonus Syndrome / diagnostic imaging
  • Opsoclonus-Myoclonus Syndrome / drug therapy
  • Radionuclide Imaging
  • Retrospective Studies
  • Speech Disorders / etiology
  • Statistics, Nonparametric
  • Steroids / therapeutic use
  • Young Adult


  • Immunoglobulins
  • Steroids