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Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Intermediate-Risk Neuroblastoma


Survival of patients with INSS stage 4 disease is strongly dependent on age. Children younger than 1 year at diagnosis have a good chance of long-term survival (i.e., a 5-year disease-free survival rate of 50%–80%),[11,12] with outcome particularly dependent on MYCN amplification and tumor cell ploidy (e.g., hyperdiploidy confers a favorable prognosis while diploidy predicts early treatment failure).[3,13] Infants aged18 months and younger at diagnosis with INSS stage 4 neuroblastoma who do not have MYCN gene amplification are categorized as intermediate risk.[14,15,16,17] The need for chemotherapy in all asymptomatic infants with stage 4 disease is somewhat controversial.[18] Stage 4 and 4S infants (N = 170) aged 12 months or younger and Stage 4 asymptomatic infants (N = 14) enrolled in an International Society of Pediatric Oncology (SIOP) trial had one of the following characteristics: 1) metastases of the 4S pattern and including positive bone metastases by iodine I 131 metaiodobenzylguanidine (131 I-MIBG) or technetium bone scan without cortical bone abnormality by computer tomography (CT) scan or plain x-ray, or 2) primary tumor stage 3 with 4S metastatic pattern. These infants were observed without initial chemotherapy, and in cases with surgical risk factors, the infants were observed without resection of the primary tumor. Although three infants underwent tumor progression, all survived. Although many were eventually treated with chemotherapy at the investigator's choice, a substantial number of infants received no chemotherapy.[18]

A small, single-institution study suggested that all MYCN-nonamplified INSS stage 3 tumors may be treated with surgical resection followed by observation without chemotherapy.[19][Level of evidence: 3iiDi]

Current Clinical Trials

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with neuroblastoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.


  1. Baker DL, Schmidt ML, Cohn SL, et al.: Outcome after reduced chemotherapy for intermediate-risk neuroblastoma. N Engl J Med 363 (14): 1313-23, 2010.
  2. Castleberry RP, Kun LE, Shuster JJ, et al.: Radiotherapy improves the outlook for patients older than 1 year with Pediatric Oncology Group stage C neuroblastoma. J Clin Oncol 9 (5): 789-95, 1991.
  3. Bowman LC, Castleberry RP, Cantor A, et al.: Genetic staging of unresectable or metastatic neuroblastoma in infants: a Pediatric Oncology Group study. J Natl Cancer Inst 89 (5): 373-80, 1997.
  4. Castleberry RP, Shuster JJ, Altshuler G, et al.: Infants with neuroblastoma and regional lymph node metastases have a favorable outlook after limited postoperative chemotherapy: a Pediatric Oncology Group study. J Clin Oncol 10 (8): 1299-304, 1992.
  5. West DC, Shamberger RC, Macklis RM, et al.: Stage III neuroblastoma over 1 year of age at diagnosis: improved survival with intensive multimodality therapy including multiple alkylating agents. J Clin Oncol 11 (1): 84-90, 1993.
  6. Matthay KK, Perez C, Seeger RC, et al.: Successful treatment of stage III neuroblastoma based on prospective biologic staging: a Children's Cancer Group study. J Clin Oncol 16 (4): 1256-64, 1998.
  7. Perez CA, Matthay KK, Atkinson JB, et al.: Biologic variables in the outcome of stages I and II neuroblastoma treated with surgery as primary therapy: a children's cancer group study. J Clin Oncol 18 (1): 18-26, 2000.
  8. Matthay KK, Sather HN, Seeger RC, et al.: Excellent outcome of stage II neuroblastoma is independent of residual disease and radiation therapy. J Clin Oncol 7 (2): 236-44, 1989.
  9. Shamberger RC, Smith EI, Joshi VV, et al.: The risk of nephrectomy during local control in abdominal neuroblastoma. J Pediatr Surg 33 (2): 161-4, 1998.
  10. Hero B, Simon T, Spitz R, et al.: Localized infant neuroblastomas often show spontaneous regression: results of the prospective trials NB95-S and NB97. J Clin Oncol 26 (9): 1504-10, 2008.
  11. Paul SR, Tarbell NJ, Korf B, et al.: Stage IV neuroblastoma in infants. Long-term survival. Cancer 67 (6): 1493-7, 1991.
  12. Bowman LC, Hancock ML, Santana VM, et al.: Impact of intensified therapy on clinical outcome in infants and children with neuroblastoma: the St Jude Children's Research Hospital experience, 1962 to 1988. J Clin Oncol 9 (9): 1599-608, 1991.
  13. Look AT, Hayes FA, Shuster JJ, et al.: Clinical relevance of tumor cell ploidy and N-myc gene amplification in childhood neuroblastoma: a Pediatric Oncology Group study. J Clin Oncol 9 (4): 581-91, 1991.
  14. Schmidt ML, Lukens JN, Seeger RC, et al.: Biologic factors determine prognosis in infants with stage IV neuroblastoma: A prospective Children's Cancer Group study. J Clin Oncol 18 (6): 1260-8, 2000.
  15. Schmidt ML, Lal A, Seeger RC, et al.: Favorable prognosis for patients 12 to 18 months of age with stage 4 nonamplified MYCN neuroblastoma: a Children's Cancer Group Study. J Clin Oncol 23 (27): 6474-80, 2005.
  16. London WB, Castleberry RP, Matthay KK, et al.: Evidence for an age cutoff greater than 365 days for neuroblastoma risk group stratification in the Children's Oncology Group. J Clin Oncol 23 (27): 6459-65, 2005.
  17. George RE, London WB, Cohn SL, et al.: Hyperdiploidy plus nonamplified MYCN confers a favorable prognosis in children 12 to 18 months old with disseminated neuroblastoma: a Pediatric Oncology Group study. J Clin Oncol 23 (27): 6466-73, 2005.
  18. De Bernardi B, Gerrard M, Boni L, et al.: Excellent outcome with reduced treatment for infants with disseminated neuroblastoma without MYCN gene amplification. J Clin Oncol 27 (7): 1034-40, 2009.
  19. Modak S, Kushner BH, LaQuaglia MP, et al.: Management and outcome of stage 3 neuroblastoma. Eur J Cancer 45 (1): 90-8, 2009.

WebMD Public Information from the National Cancer Institute

Last Updated: February 25, 2014
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