Outcome of High-Risk Stage 3 Neuroblastoma With Myeloablative Therapy and 13-cis-Retinoic Acid: A Report From the Children's Oncology Group

Background. The components of therapy required for patients with INSS Stage 3 neuroblastoma and high-risk features remain controversial. Procedure. A retrospective cohort design was used to determine if intensive chemoradiotherapy with purged autologous,S bone marrow rescue (ABMT) and/or 13-cis-retinoic acid (13-cis-RA) improved outcome for patients with high-risk neuroblastoma that was not metastatic to distant sites. We identified 72 patients with INSS Stage 3 neuroblastoma enrolled between 1991 and 1996 oil file Phase 3 CCG-3891 randomized trial. Patients were analyzed on an intent-to-treat basis using a log-rank test. Results. The 5-year event-free Survival (EFS) and overall survival (OS) rates for patients with Stage 3 neuroblastoma were 55 +/- 6% and 59 +/- 69%, respectively (n = 72). Patients randomized to ABNAT (n = 20) had 5-year EFS of 65 +/- 11% and OS of 65 +/- 11%. compared to 41 +/- 11 (P=0.21) and 46 +/- 11% (P=0.23) for patients randomized to CC (n = 23), respectively. Patients randomized to 13-cis-RA (n = 23) had 5-year EFS of 70 +/- 10% and OS of 78 +/- 99% compared to 63 +/- 12% (P=0.67) and 67 +/- 121% (P=0.55) for those receiving no further therapy (n = 16), respectively. Patients randomized to both ABMT and 13-cis-RA (n=6) had a 5-year EFS of 80 +/- 11% and OS of 100%. Conclusion. Patients with high-risk Stage 3 neuroblastoma have an overall poor prognosis despite aggressive chemoradiotherapy. Further studies are warranted to determine if myeloblative consolidation followed by 13-cis-RA maintenance therapy statistically significantly improves outcome. Pediatr Blood Cancer 2009; 52:44-50. (c) 2608 Wiley-Liss, Inc.