Outcome of children with opsoclonus-myoclonus regardless of etiology

Area of Science:

• Pediatric Neurology
• Neuro-oncology
• Clinical Pediatrics

Background:

• Opsoclonus-myoclonus is a rare paraneoplastic or idiopathic neurological disorder.
• Neuroblastoma is a common underlying cause in pediatric cases.
• Developmental outcomes in affected children require further investigation.

Purpose of the Study:

• To analyze the clinical presentation, treatment outcomes, and neurodevelopmental impact of opsoclonus-myoclonus in children.
• To investigate the association between opsoclonus-myoclonus and neuroblastoma.
• To evaluate the efficacy of different treatment modalities.

Main Methods:

• Retrospective case series of 11 pediatric patients with opsoclonus-myoclonus.
• Review of medical records, including neuroblastoma screening, treatment history (ACTH, prednisone), and neurodevelopmental assessments.
• Comparison of outcomes between children with and without neuroblastoma.

Main Results:

• Eight of 11 patients had occult neuroblastoma.
• Eight patients experienced delayed development, motor incoordination, and speech delay.
• ACTH treatment was associated with frequent symptom recurrence upon dose reduction or discontinuation; prednisone was ineffective.
• Tumor removal did not improve symptoms in neuroblastoma-associated cases.
• Children without neuroblastoma showed a better prognosis for normal neurologic development (2/3 vs. 1/8).

Conclusions:

• Opsoclonus-myoclonus in children, particularly when associated with neuroblastoma, frequently results in significant and persistent developmental delays.
• Current treatment strategies, including ACTH, have limited success in preventing symptom recurrence.
• The absence of neuroblastoma appears to correlate with a more favorable neurodevelopmental outcome, suggesting distinct pathophysiological pathways or prognoses.