Research Papers:

MYCN-amplified stage 2/3 neuroblastoma: excellent survival in the era of anti-GD2 immunotherapy

Brian H. Kushner _, Michael P. LaQuaglia, Shakeel Modak, Suzanne L. Wolden, Ellen M. Basu, Stephen S. Roberts, Kim Kramer, Karima Yataghene, Irene Y. Cheung and Nai-Kong V. Cheung

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Oncotarget. 2017; 8:95293-95302. https://doi.org/10.18632/oncotarget.20513

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Brian H. Kushner1, Michael P. LaQuaglia1, Shakeel Modak1, Suzanne L. Wolden2, Ellen M. Basu1, Stephen S. Roberts1, Kim Kramer1, Karima Yataghene1, Irene Y. Cheung1 and Nai-Kong V. Cheung1

1Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA

2Department of Radiation Oncology, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA

Correspondence to:

Brian H. Kushner, email: [email protected]

Keywords: neuroblastoma; anti-GD2 antibody; autologous transplantation; cytokine; MYCN amplification

Received: May 07, 2017    Accepted: August 07, 2017    Published: August 24, 2017


High-risk neuroblastoma (HR-NB) includes MYCN-amplified stage 2/3, but reports covering anti-GD2 immunotherapy, which recently became standard for HR-NB, do not provide details on this subset. We now report on all 20 MYCN-amplified stage 2/3 patients who received induction chemotherapy at our center during the era of consolidation with anti-GD2 antibody 3F8/ granulocyte-macrophage colony-stimulating factor (GM-CSF) (2000-2015). Early in this period, consolidation included autologous stem-cell transplantation (ASCT). Event-free survival (EFS) and overall survival (OS) were estimated using Kaplan-Meier analyses.

With induction, 19/20 (95%) patients achieved complete/very good partial remission (CR/VGPR) but one had progressive disease with early death. One responder did not receive consolidation and died of relapse. Five-year post-diagnosis EFS/OS rates for all 20 patients were 72%/84%. The 18 CR/VGPR patients who received consolidation had EFS/OS 81%/94% at five years from starting 3F8/GM-CSF: 4/4 ASCT patients remained relapse-free, while 11/14 non-ASCT patients remained relapse-free and two of the three relapsed patients achieved 2nd CR (consolidated by retreatment with 3F8/GM-CSF) and remained in 2nd CR at 36+ and 95+ months post-relapse. The 14 non-ASCT patients had EFS/OS 73.5%/93% at five years from starting 3F8/GM-CSF.

This subset appears to have a good prognosis with contemporary multi-modality therapy, possibly even without ASCT.

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