Long-term outcomes in patients with stage IV neuroblastoma

Long-term outcome studies in survivors with stage IV neuroblastoma (NB) are sparse. This review evaluates late complications and long-term outcomes in stage IV NB survivors. A retrospective review of stage IV NB survivors was performed to analyze outcomes, including long-term morbidity, recurrence, and survival. Of 153 patients with stage IV NB, 52 (34%) survived (male-female, 26:26). Age at diagnosis was 29.1 ± 31.7 months in survivors. Eighteen were 1 year or younger and 34 were older than 1 year compared with 10 nonsurvivors 1 year or younger and 91 older than 1 year ( P = .0003, Fisher's Exact test). Primary tumor sites were adrenal (35), retroperitoneal (11), mediastinal (3), pelvic (2), and no primary with tumor metastases identified (1). Ten survivors had favorable and 16 had unfavorable histology compared with 1 favorable and 18 unfavorable in nonsurvivors ( P = .01). Four survivors had MYCN amplification (≥10 copies) and 2 deletions of 1p and 11q. Sites of metastasis in survivors and nonsurvivors were similar. Treatment in survivors included surgery in 51 (75% [39/51] complete tumor resection [CTR]); chemotherapy, 50; radiation, 17; stem cell transplantation, 20; and bone marrow transplant, 1. In nonsurvivors, 13 (25%) of 53 ( P ≪ .0001) had CTR, 18 stem cell transplantation, and 12 bone marrow transplant. Six patients had tumor recurrence but survived (mean, 9.3 ± 8.3 years; range, 6 months–24 years). Recurrence was local (1), distant (2), and both (3) and was treated by resection, chemotherapy, and radiation. The mean age of survivors was 12.4 ± 8.3 years (range, 2-34 years). In all stage IV cases, event-free survival was 30% and overall survival was 34%. Long-term complications occurred in 23 (44%) survivors, including endocrine disturbances (7), orthopedic (5), cataracts (2), adhesive bowel obstruction (2), hypertension (1), bronchiolitis (1), blindness (1), peripheral neuropathy (1), nonfunctioning kidney (1), cholelithiasis (1), and thyroid nodule (1). Only 34% of patients with stage IV NB survived despite aggressive multimodal therapy. Age of younger than 1 year, favorable pathology, CTR, and no recurrence were the only statistically significant factors that favored survival. Forty-four percent of survivors experienced late morbidity, and tumor recurred in 6 (11.5%) of 52. Patients should be monitored for tumor recurrence and long-term sequelae. New methods of treatment are required to achieve better outcomes.