Abstract
Background
Stage 4 and MYCN amplified (MNA) neuroblastoma in children have a poor prognosis. Our aim was to increase initial and long‐term reponse in this population.
Procedure
High‐risk children were studied according to the International Neuroblastoma Staging System, then treated with high‐dose cyclophosphamide and high‐dose carboplatin, followed by surgery and autologous stem cell transplant or maintenance chemotherapy.
Results
From June 1992 to December 1998, 83 children were admitted in the study (72 stage 4> 1 year, 5 stage 4 MNA infants, and 6 MNA stage 3 children); tumor tissue was obtained from 73, MYCN was performed in 65, being amplified in 21 (32%). Induction chemotherapy was administered in the expected time in 35% of patients. Its toxicity was mainly hematologic followed by infections, and there were 3 chemotherapy‐related deaths. Delayed surgery was performed on 60 patients with complete or >90% resection in 80% of cases. Chemotherapy plus surgery produced some response in 90% of patients, 53% were in CR/VGPR; 49 children received autologous SCT, and 16 received maintenance chemotherapy for 9 months. Follow‐up ranges are 1–87 months, mean 30 months. S and EFS at 4 years are 0.33 (SD 0.02).
Conclusions
High‐dose cyclophosphamide and high‐dose carboplatin are effective in the initial treatment of neuroblastoma; combined with surgery they produce some response in most patients. Nevertheless, the CR/VGPR rate reaches only 53%. Survival time has also been prolonged but most patients relapse with metastases. Med Pediatr Oncol 2001;37:537–542. © 2001 Wiley‐Liss, Inc.