• Efficacy as measured by assessment of response of opsoclonus, gait, neural, stance, arm and hand function at baseline, 2 months, 6 months, and 1 year [ Designated as safety issue: No ]
  
• Response as measured by assessment of opsoclonus, gait, neural, stance, arm and hand function at baseline, 2 months, 6 months, and 1 year [ Designated as safety issue: No ]
  

• Motor coordination as assessed by neurological examination and Vineland Adaptive Behavior Scale at baseline, 1 and 5 years [ Designated as safety issue: No ]
  
• Functional outcome as assessed by age-appropriate neuropsychological testing at baseline, 1 and 5 years [ Designated as safety issue: No ]
  
• Biology of neuroblastoma associated opsoclonus-myoclonus-ataxia syndrome, specifically by MRI findings, anti-neuronal antibodies, SCF findings and tumor biology at baseline, 6 months, and 1 year [ Designated as safety issue: No ]
  
• Long-term prognosis for neurologic recovery by neurological examination at baseline, 1 and 5 years [ Designated as safety issue: No ]
  
• Tumor outcome in terms of event-free survival and overall survival at 1, 5, and 10 years [ Designated as safety issue: No ]
  

OBJECTIVES:

• Determine whether cyclophosphamide and prednisone with or without immune globulin is a reasonable baseline standard therapy for pediatric patients with neuroblastoma-associated opsoclonus-myoclonus-ataxia (OMA) syndrome.
• Determine whether immunosuppressive therapy with cyclophosphamide and prednisone is an effective backbone therapy for OMA upon which to build additional treatment for these patients.
• Determine whether these regimens improve OMA syndrome in these patients.
• Determine whether these regimens improve motor coordination in these patients.
• Determine whether these regimens improve functional outcome in these patients.
• Compare the event-free and overall survival of patients treated with these regimens.

OUTLINE: This is a randomized, multicenter study. Patients are stratified according to risk group per protocol COG-ANBL00B1 (low risk vs intermediate risk on COG-A3961 vs high risk on COG-A3973).

• Chemotherapy: Patients with intermediate-risk or high-risk neuroblastoma receive chemotherapy (including cyclophosphamide) according to the standard of care for the stage of primary neuroblastoma, beginning on day 0.

Patients with low-risk neuroblastoma (and not receiving other chemotherapy) receive cyclophosphamide IV over 1 hour on day 0. Treatment repeats every 4 weeks for 6 courses in the absence of disease progression or unacceptable toxicity.

All patients receive prednisone twice daily for 3 months and then every other day for 7-15 months.

• Immune globulin therapy: Patients are randomized to 1 of 2 treatment arms.

  • Arm I: Patients receive immune globulin IV on days -2 and -1, at weeks 4, 8, 12, 16, 20, and 24, and then at months 8, 10, and 12 after therapy. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients with no response after 6 months go off treatment.
  • Arm II: Patients do not receive immune globulin. Patients with unresponsive opsoclonus-myoclonus-ataxia syndrome after 2 months or progression after 6 months may cross over to arm I.

Patients are followed during therapy every month for 6 months, at 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 18-52 patients (9-26 per treatment arm) will be accrued for this study within 2-5.8 years.