Trial Information

COOPERATIVE MULTICENTER TRIAL FOR THE TREATMENT OF INFANTS WITH NEUROBLASTOMA

OBJECTIVES: I. Determine the incidence of spontaneous regression of localized neuroblastoma
in infants. II. Determine how many infants with neuroblastoma do not need chemotherapy. III.
Evaluate the time course of regression by radiologic criteria and catecholamine metabolites.
IV. Determine the reliability of risk estimation by molecular characteristics (N-myc
amplification, CD44, del 1p) compared to clinical criteria. V. Evaluate whether reduced
therapeutic toxicity results in a decrease in treatment-related deaths. VI. Correlate
cytostatic drug levels with chemotherapy side effects.

OUTLINE: All patients undergo resection of the primary tumor and N-myc determination within
6 months of age, unless critically ill, then are treated according to risk. Patients with
amplified N-myc or with indeterminate N-myc amplification but with other risk features are
treated per protocol GER-NB90. Stage 4S patients who are critically ill or thrombocytopenic
receive doxorubicin, vincristine, and cyclophosphamide over 7 days. Patients with no
amplification of N-myc are observed for 6 months (until between 12 and 18 months of age).
Patients with minimal residual disease (less than 10% or diameter no greater than 2-5 mm)
continue observation, while those with residual disease but no disease progression undergo
repeat biopsy. Patients whose biopsy indicates tumor regression also continue observation.
All other patients, including those with disease progression, are treated per protocol
GER-NB90.

PROJECTED ACCRUAL: 36-44 patients per year will be accrued (22-27 patients with stages 1-3,
8-10 patients with stage 4S, and 6-7 patients with stage 4 neuroblastoma).