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Outcome of high‐risk stage 3 neuroblastoma with myeloablative therapy and 13‐ cis ‐retinoic acid: A report from the Children's Oncology Group
Author(s) -
Park Julie R.,
Villablanca Judith G.,
London Wendy B.,
Gerbing Robert B.,
HaasKogan Daphne,
Adkins E. Stanton,
Attiyeh Edward F.,
Maris John M.,
Seeger Robert C.,
Reynolds C. Patrick,
Matthay Katherine K.
Publication year - 2009
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21784
Subject(s) - medicine , neuroblastoma , randomized controlled trial , oncology , stage (stratigraphy) , chemoradiotherapy , chemotherapy , paleontology , genetics , biology , cell culture
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 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 on the 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 ± 6%, respectively (n = 72). Patients randomized to ABMT (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 ± 9% compared to 63 ± 12% ( P = 0.67) and 67 ± 12% ( 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 myeloablative consolidation followed by 13‐ cis ‐RA maintenance therapy statistically significantly improves outcome. Pediatr Blood Cancer 2009;52:44–50. © 2008 Wiley‐Liss, Inc.