Neuroblastoma Treatment–for health professionals (PDQ®)

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Changes to this Summary (12/01/2015)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

General Information About Neuroblastoma

Added text to state that although most neuroblastoma tumors are initially sensitive to chemotherapy, many relapse in local and/or metastatic sites. Modern genetic analysis, including deep whole-genome sequencing of primary and relapsed neuroblastomas from the same patients, revealed many new mutations and complex clonal evolution of pre-existing minor subclones. The most common new mutations found were in the RAS-MAPK pathway (cited Eleveld et al. and Schramm et al. as references 29 and 30, respectively).

Cellular Classification of Neuroblastic Tumors

Added Teshiba et al. as reference 6.

Stage Information for Neuroblastoma

Revised text to state that the International Neuroblastoma Risk Group Staging System (INRGSS) has predictive value for patients with lower-stage disease, with a 5-year event-free survival (EFS) of 90% and overall survival (OS) of 98% for stage L1 patients, compared with an EFS of 79% and OS of 89% for stage L2 patients. Also added text to state that among INRG stage L2 children, International Neuroblastoma Staging System (INSS) stage 2 patients do significantly better than do INSS stage 3 patients in 5-year OS (cited Monclair et al. as reference 24).

Treatment Option Overview for Neuroblastoma

Added text to state that a meta-analysis of stage 3 versus stage 4 neuroblastoma patients, at all ages combined, found an advantage for gross-total resection over subtotal resection in stage 3 neuroblastoma only, not stage 4 (cited Mullassery et al. as reference 21). Also added text to state that a small study suggested that after neoadjuvant chemotherapy, completeness of resection was affected by the number of image-defined risk factors remaining (cited Irtan et al. as reference 22).

Treatment of High-Risk Neuroblastoma

Revised text about the randomized study that was performed to compare high-dose therapy with purged autologous bone marrow transplant (ABMT) to clarify the randomization and to update the survival statistics.

Added text to state that an updated Cochrane review evaluated three randomized clinical trials comparing ABMT with standard chemotherapy. EFS was significantly better for ABMT, but there was no statistically significant difference in OS (cited Yalçin et al. as reference 20).

Added text to state that the ch14.18 antibody has been approved by the U.S. Food and Drug Administration.

Recurrent Neuroblastoma

Added text to state that modern comprehensive molecular analysis comparing primary and relapsed neuroblastoma from the same patients revealed extensive clonal enrichment and several new mutations, with many tumors showing new or clonal-enriched mutations in the RAS-MAPK pathway. This was true for patients with both high-risk and low-risk tumors at diagnosis (cited Eleveld et al. and Schramm et al. as references 3 and 4, respectively).

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® - NCI's Comprehensive Cancer Database pages.

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