Biologic variables in the outcome of stages I and II neuroblastoma treatedwith surgery as primary therapy: A children's cancer group study

Purpose: To determine prospectively whether surgery alone is sufficient the rapy for Evens stages I and II neuroblastoma and to define biologic and cli nical features having prognostic potential for this group. Patients and Methods: Between June 1989 and August 1995, 374 eligible child ren (age range, 0 to 18 years) with newly diagnosed stage I (n = 141) and s tage II (n = 233) neuroblastoma were registered onto Children's Cancer Grou p trial 3881. Surgical resection was the only primary therapy except in cas es with spinal cord compression, where radiation therapy was allowed. Event -free survival (EFS) and overall survival (OS) were analyzed by life-table methods according to clinical and biologic features. Results: EFS and OS (mean +/- SE) for all stage I patients were 93% +/- 3.0 % and 99% +/- 1.0%, respectively, compared with 81% +/- 4.0% and 98% +/- 2. 0%, respectively, for stage II patients. The significantly higher recurrenc e rate among stage II patients was managed successfully in 38 of 43 childre n with either surgery or multimodality treatment. There was one death among stage I patients and six among stage II. For stage II patients tumor MYCN gene amplication, unfavorable histopathology, an age greater than 2 years, and positive lymph nodes predicted a lower OS (P < .05). Conclusion: Children with stages I and II neuroblastoma have 98% survival w ith surgery alone as primary therapy. Supplemental treatment was necessary in only 10% of stage I patients and 20% of stage II patients. In children w ith localized neuroblastoma, a subset of patients that are at higher risk f or death can be defined as those with stage II disease who have tumor MYCN amplification or who are greater than or equal to 2 years of age with eithe r unfavorable histopathology or positive lymph nodes. (C) 2000 by American Society of Clinical Oncology.