Striking dichotomy in outcome of MYCN-amplified neuroblastoma in the contemporary era Journal Article
| Authors: | Kushner, B. H.; Modak, S.; Kramer, K.; LaQuaglia, M. P.; Yataghene, K.; Basu, E. M.; Roberts, S. S.; Cheung, N. K. V. |
| Article Title: | Striking dichotomy in outcome of MYCN-amplified neuroblastoma in the contemporary era |
| Abstract: | BACKGROUND The authors exploited a large database to investigate the outcomes of patients with high-risk neuroblastoma in the contemporary era. METHODS All patients with high-risk neuroblastoma aged <12 years who were treated during induction at the authors' institution from 2000 through 2011 were studied, including 118 patients with MYCN-amplified [MYCN(+)] disease and 127 patients aged >18 months with MYCN-nonamplified [MYCN(-)] stage 4 disease. RESULTS A complete response/very good partial response (CR/VGPR) to induction was correlated with significantly superior event-free survival (EFS) (P<.001) and overall survival (OS) (P<.001) compared with a partial response or less. Patients with MYCN(+) and MYCN(-) disease had similar rates of CR/VGPR to induction (P=.366), and those with MYCN(+) and MYCN(-) disease who attained a CR/VGPR had similar EFS (P=.346) and OS (P=.542). In contrast, only MYCN(+) patients had progressive disease as a response to induction (P<.001), and early death from progressive disease (<366 days after diagnosis) was significantly more common (P<.001) among those with MYCN(+) disease. Overall, among patients who had a partial response or less, MYCN(+) patients had significantly inferior EFS (P<.001) and OS (P<.001) compared with MYCN(-) patients, which accounted for the significantly worse EFS (P=.008) and OS (P=.002) for the entire MYCN(+) cohort versus the MYCN(-) cohort. CONCLUSIONS Patients with MYCN(-), high-risk neuroblastoma display a broad, continuous spectrum with regard to response and outcome, whereas MYCN(+) patients either have an excellent response to induction associated with good long-term outcome or develop early progressive disease with a poor outcome. This extreme dichotomy in the clinical course of MYCN(+) patients points to underlying biologic differences with MYCN(+) neuroblastoma, the elucidation of which may have far-reaching implications, including improved risk classification at diagnosis and the identification of targets for treatment. Cancer 2014;120:2050-2059. © 2014 American Cancer Society. |
| Keywords: | neuroblastoma; induction; mycn; contemporary therapy |
| Journal Title: | Cancer |
| Volume: | 120 |
| Issue: | 13 |
| ISSN: | 0008-543X |
| Publisher: | Wiley Blackwell |
| Date Published: | 2014-07-01 |
| Start Page: | 2050 |
| End Page: | 2059 |
| Language: | English |
| DOI: | 10.1002/cncr.28687 |
| PROVIDER: | scopus |
| PUBMED: | 24691684 |
| PMCID: | PMC4326052 |
| DOI/URL: | |
| Notes: | Cancer -- Export Date: 8 July 2014 -- CODEN: CANCA -- Source: Scopus |
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