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Neuroblastoma Treatment (PDQ®)

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Changes to this Summary (12/15/2014)

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 Smith et al. as reference 1.

Added text to state that for neuroblastoma, the 5-year survival rate increased between 1975 and 2010, from 86% to 95% for children younger than 1 year and from 34% to 68% for children aged 1 to 14 years.

Added text to state that in North American clinical trials reported in the 1990s, infants aged 1 year and younger had a cure rate higher than 80%, while older children had a cure rate of 50% to 70% with then-current, relatively intensive therapy (cited 1991 Castleberry et al., 1992 Castleberry et al., and West et al. as references 55, 57, and 58, respectively).

Revised text to state that neuroblastoma has a worse long-term prognosis in an adolescent older than 10 years or in an adult than in a child, regardless of stage or site; and, in many cases, it has a more prolonged course when treated with standard doses of chemotherapy. Also added text to state that although these patients may have a more indolent course and infrequent MYCN amplification, older children with advanced disease have a poor rate of survival (cited Mossé et al. as reference 66).

Stage Information for Neuroblastoma

Added text to state that a study from the International Neuroblastoma Risk Group (INRG) database found 146 patients with distant metastases limited to lymph nodes, termed stage 4N, who tended to have favorable-biology disease and a good outcome, which suggests that less-intensive therapy might be considered (cited Morgenstern et al. as reference 22).

Revised text to state that image-defined risk factors (IDRFs) are surgical risk factors, detected by imaging, which could potentially make total tumor excision risky or difficult at the time of diagnosis and increase the risk of surgical complications.

Added text to state that the Children's Oncology Group (COG) has been collecting and evaluating International Neuroblastoma Risk Group Staging System (INRGSS) data since 2006. A COG trial that opened in 2014 uses the INRGSS to determine therapy for patients with certain localized disease and stage 4S patients. Note that the International Neuroblastoma Staging System (INSS) allows patients up to age 12 months in stage 4S, while the INRGSS allows patients up to age 18 months in stage MS.

Added text to list the IDRFs.

Treatment Option Overview for Neuroblastoma

Revised text to state that most children in North America were treated according to the COG risk-group assignment, even if they were not enrolled in a COG study. Also added text to state that in the most recent COG study, the INRG system was used to assign treatment; in the INRG system, each child is assigned to a group on the basis of image-defined potential surgical risk, age, and the presence or absence of metastasis.

Added text to state that in 2012, the COG Neuroblastoma Committee defined favorable genomics, for purposes of risk assignment, as hyperdiploid neuroblastoma cells without segmental copy number aberrations, including no loss of copy number at 1p, 3p, 4p, or 11q and no gain of copy number at 1q, 2p, or 17q.

Added text to state that in recent studies, select patients have been observed without undergoing chemotherapy or attempted resection.

Added text about the treatment of L1 and L2 tumors (cited Hero et al. as reference 15).

Added text to state that according to the surgical guidelines described in the recent intermediate-risk neuroblastoma clinical trial, the primary tumor is not routinely resected in patients with 4S neuroblastoma.

Treatment of Low-Risk Neuroblastoma

Added text to state that similar outcomes were seen in a nonrandomized clinical trial in Japan (cited Iehara et al. as reference 4).

Added Treatment Options Under Clinical Evaluation as a new subsection.

Treatment of Intermediate-Risk Neuroblastoma

Added text to state that a nonrandomized clinical trial in Japan also reported excellent outcomes for infants with stage 3 neuroblastoma without MYCN amplification (cited Iehara et al. as reference 5).

Added Treatment Options Under Clinical Evaluation as a new subsection.

Treatment of High-Risk Neuroblastoma

Added text to state that a study from the INRG database found 146 patients with distant metastases limited to lymph nodes, termed stage 4N, who tended to have favorable-biology disease and a good outcome, which suggests that for this special subgroup of high-risk, stage 4 patients, less-intensive therapy might be considered (cited Morgenstern et al. as reference 5).

Added text to state that a review of 147 allogeneic transplant cases submitted to the Center for International Blood and Marrow Transplant Research found no advantage for allogeneic transplant over autologous transplant, even if the allogeneic transplant recipient had received a previous autologous transplant (cited Hale et al. as reference 20).

Treatment of Stage 4S Neuroblastoma

Added Treatment Options Under Clinical Evaluation as a new subsection.

Recurrent Neuroblastoma

Added text to state that in neuroblastoma, subclonal ALK mutations may be present at diagnosis, with subsequent clonal expansion at relapse; consequently, serial sampling of progressive tumors may be revealing (cited Schleiermacher et al. as reference 2).

Added text to state that allogeneic transplantation has a historically low success rate in recurrent or progressive neuroblastoma. In a retrospective registry study, allogeneic stem cell transplant (SCT) after a previous autologous SCT appeared to offer minimal benefit. Disease recurrence remains the most common cause of treatment failure (cited Hale et al. as reference 24).

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.

  • Updated: December 15, 2014