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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 JR, Villablanca JG, London WB, Gerbing RB, Haas-Kogan D, Adkins ES, Attiyeh EF, Maris JM, Seeger RC, Reynolds CP, Matthay KK, Children's Oncology Group

Affiliation(s): Department of Pediatrics, Children's Hospital and Regional Medical Center, University of Washington, Seattle, Washington 98105, USA. julie.park@seattlechildrens.org

Publication date & source: 2009-01, Pediatr Blood Cancer., 52(1):44-50.

Publication type: Clinical Trial, Phase III; Comparative Study; Randomized Controlled Trial; Research Support, N.I.H., Extramural

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.

Page last updated: 2009-02-08

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