Highlights & Basics
- Neuroblastoma is a rare, malignant tumor arising from the embryological neural crest element of the peripheral sympathetic nervous system.
- The most common extracranial solid tumor in children; the majority of patients are diagnosed by 5 years of age.
- Diagnosis can usually be confirmed by urine catecholamines and imaging; however, biopsy of the primary site (or bone marrow for staging purposes) is required to establish biology (i.e., cytogenetics, pathology) and risk stratification.
- Treatment depends primarily on risk classification and varies from observation alone for certain low-risk patients to intense multimodal therapy for high-risk patients.
- Prognosis is excellent for patients with low-risk disease and poor for those with high-risk disease.
Quick Reference
History & Exam
Key Factors
Other Factors
Diagnostics Tests
Treatment Options
Definition
Epidemiology
Etiology
Pathophysiology
Images
Neural crest development and neuroblastoma formation; EMT = epithelial to mesenchymal transition
CT scan of the abdomen showing paraspinal neuroblastoma
CT scan showing a thoracic paraspinal neuroblastoma wrapping around the spine
123-iodine-metaiodobenzylguanidine (MIBG) scintigraphy showing multifocal bony metastases
Citations
Brisse HJ, McCarville MB, Granata C, et al. Guidelines for imaging and staging of neuroblastic tumors: consensus report from the International Neuroblastoma Risk Group Project. Radiology. 2011 Oct;261(1):243-57.[Abstract][Full Text]
Shimada H, Ambros IM, Dehner LP, et al. The international neuroblastoma pathology classification (the Shimada system). Cancer. 1999 Jul 15;86(2):364-72.[Abstract]
Brodeur GM, Pritchard J, Berthold F, et al. Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment. J Clin Oncol. 1993 Aug;11(8):1466-77.[Abstract]
1. Brodeur G, Hogarty M, Bagatell R, et al. Neuroblastoma. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:772.
2. Brisse HJ, McCarville MB, Granata C, et al. Guidelines for imaging and staging of neuroblastic tumors: consensus report from the International Neuroblastoma Risk Group Project. Radiology. 2011 Oct;261(1):243-57.[Abstract][Full Text]
3. Angstman KB, Miser JS, Franz WB 3rd. Neuroblastoma. Am Fam Physician. 1990 Jan;41(1):238-44.[Abstract]
4. Shimada H, Ambros IM, Dehner LP, et al. The international neuroblastoma pathology classification (the Shimada system). Cancer. 1999 Jul 15;86(2):364-72.[Abstract]
5. Pollard ZF, Greenberg MF, Bordenca M, et al. Atypical acquired pediatric Horner syndrome. Arch Ophthalmol. 2010 Jul;128(7):937-40.[Abstract][Full Text]
6. Ogita S, Tokiwa K, Takahashi T, et al. Congenital cervical neuroblastoma associated with Horner syndrome. J Pediatr Surg. 1988 Nov;23(11):991-2.[Abstract]
7. Brunklaus A, Pohl K, Zuberi SM, et al. Investigating neuroblastoma in childhood opsoclonus-myoclonus syndrome. Arch Dis Child. 2012 May;97(5):461-3.[Abstract][Full Text]
8. Kaplan SJ, Holbrook CT, McDaniel HG, et al. Vasoactive intestinal peptide secreting tumors of childhood. Am J Dis Child. 1980 Jan;134(1):21-4.[Abstract]
9. Ward E, DeSantis C, Robbins A, et al. Childhood and adolescent cancer statistics, 2014. CA Cancer J Clin. 2014 Mar-Apr;64(2):83-103.[Abstract][Full Text]
10. Stiller CA, Parkin DM. International variations in the incidence of neuroblastoma. Int J Cancer. 1992 Oct 21;52(4):538-43.[Abstract]
11. Maris JM, Chatten J, Meadows AT, et al. Familial neuroblastoma: a three-generation pedigree and a further association with Hirschsprung disease. Med Pediatr Oncol. 1997 Jan;28(1):1-5.[Abstract]
12. Kushner BH, Gilbert F, Helson L. Familial neuroblastoma: case reports, literature review, and etiologic considerations. Cancer. 1986 May 1;57(9):1887-93.[Abstract]
13. Shahar E, Shinawi M. Neurocristopathies presenting with neurologic abnormalities associated with Hirschsprung's disease. Pediatr Neurol. 2003 May;28(5):385-91.[Abstract]
14. Nemecek ER, Sawin RW, Park J. Treatment of neuroblastoma in patients with neurocristopathy syndromes. J Pediatr Hematol Oncol. 2003 Feb;25(2):159-62.[Abstract]
15. Stovroff M, Dykes F, Teague WG. The complete spectrum of neurocristopathy in an infant with congenital hypoventilation, Hirschsprung's disease, and neuroblastoma. J Pediatr Surg. 1995 Aug;30(8):1218-21.[Abstract]
16. Kushner BH, Hajdu SI, Helson L. Synchronous neuroblastoma and von Recklinghausen's disease: a review of the literature. J Clin Oncol. 1985 Jan;3(1):117-20.[Abstract]
17. Blatt J, Olshan AF, Lee PA, et al. Neuroblastoma and related tumors in Turner's syndrome. J Pediatr. 1997 Nov;131(5):666-70.[Abstract]
18. DuBois SG, Kalika Y, Lukens JN, et al. Metastatic sites in stage IV and IVS neuroblastoma correlate with age, tumor biology, and survival. J Pediatr Hematol Oncol. 1999 May-Jun;21(3):181-9.[Abstract]
19. Louis CU, Shohet JM. Neuroblastoma: molecular pathogenesis and therapy. Annu Rev Med. 2015;66:49-63.[Abstract][Full Text]
20. Sadilkova K, Dugaw K, Benjamin D, et al. Analysis of vanillylmandelic acid and homovanillic acid by UPLC-MS/MS in serum for diagnostic testing for neuroblastoma. Clin Chim Acta. 2013 Sep 23;424:253-7.[Abstract]
21. LaBrosse EH, Com-Nougué C, Zucker JM, et al. Urinary excretion of 3-methoxy-4-hydroxymandelic acid and 3-methoxy-4-hydroxyphenylacetic acid by 288 patients with neuroblastoma and related neural crest tumors. Cancer Res. 1980 Jun;40(6):1995-2001.[Abstract][Full Text]
22. Cangemi G, Barco S, Reggiardo G, et al. Interchangeability between 24-hour collection and single spot urines for vanillylmandelic and homovanillic acid levels in the diagnosis of neuroblastoma. Pediatr Blood Cancer. 2013 Dec;60(12):E170-2.[Abstract]
23. Brodeur GM, Pritchard J, Berthold F, et al. Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment. J Clin Oncol. 1993 Aug;11(8):1466-77.[Abstract]
24. Hawthorne HC Jr, Nelson JS, Witzleben CL, et al. Blanching subcutaneous nodules in neonatal neuroblastoma. J Pediatr. 1970 Aug;77(2):297-300.[Abstract]
25. Scanga DR, Martin WH, Delbeke D. Value of FDG PET imaging in the management of patients with thyroid, neuroendocrine, and neural crest tumors. Clin Nucl Med. 2004 Feb;29(2):86-90.[Abstract]
26. Matthay KK, Edeline V, Lumbroso J, et al. Correlation of early metastatic response by 123I-metaiodobenzylguanidine scintigraphy with overall response and event-free survival in stage IV neuroblastoma. J Clin Oncol. 2003 Jul 1;21(13):2486-91.[Abstract]
27. Kushner BH, Yeung HW, Larson SM, et al. Extending positron emission tomography scan utility to high-risk neuroblastoma: fluorine-18 fluorodeoxyglucose positron emission tomography as sole imaging modality in follow-up of patients. J Clin Oncol. 2001 Jul 15;19(14):3397-405.[Abstract]
28. Boubaker A, Bischof Delaloye A. Nuclear medicine procedures and neuroblastoma in childhood. Their value in the diagnosis, staging and assessment of response to therapy. Q J Nucl Med. 2003 Mar;47(1):31-40.[Abstract][Full Text]
29. Kushner BH, Kramer K, LaQuaglia MP, et al. Neuroblastoma in adolescents and adults: the Memorial Sloan-Kettering experience. Med Pediatr Oncol. 2003 Dec;41(6):508-15.[Abstract]
30. Fernandez C, Geller J, Ehrlich P, et al. Renal tumors. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:753.
31. Hawkins D, Brennan B, Bolling T, et al. Ewing sarcoma. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:855.
32. Wexler L, Skapek S, Helman L. Rhabdomyosarcoma. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:798
33. Meyers R, Trobaugh-Lotario A, Malogolowkin M, et al. Liver tumors. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:726.
34. Rabin K, Gramatges M, Margolin J, et al. Acute lymphoblastic leukemia. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:463.
35. Metzger M, Krasin M, Choi J, et al. Hodgkin lymphoma. In: Pizzo P, Poplack D, eds. Principles and practice of pediatric oncology. Philidelphia, PA: Lippincott Williams & Wilkins; 2016:568.
36. Khuri FJ, Alton DJ, Hardy BE, Cook GT, Churchill BM. Adrenal hemorrhage in neonates: report of 5 cases and review of the literature. J Urol. 1980 Nov;124(5):684-7.[Abstract]
37. Vukelic D, Bozinovic D, Morovic M, et al. Opsoclonus-myoclonus syndrome in a child with neuroborreliosis. J Infect. 2000 Mar;40(2):189-91.[Abstract]
38. Sheth RD, Horwitz SJ, Aronoff S, et al. Opsoclonus myoclonus syndrome secondary to Epstein-Barr virus infection. J Child Neurol. 1995 Jul;10(4):297-9.[Abstract]
39. Ertekin V, Tan H. Opsoclonus-myoclonus syndrome attributable to hepatitis C infection. Pediatr Neurol. 2010 Jun;42(6):441-2.[Abstract]
40. Cardesa-Salzmann TM, Mora J, García Cazorla MA, et al. Epstein-Barr virus related opsoclonus-myoclonus-ataxia does not rule out the presence of occult neuroblastic tumors. Pediatr Blood Cancer. 2006 Dec;47(7):964-7.[Abstract]
41. Candler PM, Dale RC, Griffin S, et al. Post-streptococcal opsoclonus-myoclonus syndrome associated with anti-neuroleukin antibodies. J Neurol Neurosurg Psychiatry. 2006 Apr;77(4):507-12.[Abstract][Full Text]
42. Yamamoto K, Ohta S, Ito E, et al. Marginal decrease in mortality and marked increase in incidence as a result of neuroblastoma screening at 6 months of age: cohort study in seven prefectures in Japan. J Clin Oncol. 2002 Mar 1;20(5):1209-14.[Abstract]
43. Woods WG, Gao RN, Shuster JJ, et al. Screening of infants and mortality due to neuroblastoma. N Engl J Med. 2002 Apr 4;346(14):1041-6.[Abstract][Full Text]
44. Schilling FH, Spix C, Berthold F, et al. Neuroblastoma screening at one year of age. N Engl J Med. 2002 Apr 4;346(14):1047-53.[Abstract][Full Text]
45. Chamberlain J. Screening for neuroblastoma: a review of the evidence. J Med Screen. 1994 Jul;1(3):169-75.[Abstract]
46. Park JR, Bagatell R, Cohn SL, et al. Revisions to the International Neuroblastoma Response Criteria: a consensus dtatement from the National Cancer Institute Clinical Trials Planning Meeting. J Clin Oncol. 2017 Aug 1;35(22):2580-7.[Abstract][Full Text]
47. 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. 2005 Sep 20;23(27):6474-80.[Abstract][Full Text]
48. 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. 2005 Sep 20;23(27):6459-65.[Abstract][Full Text]
49. 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. 2005 Sep 20;23(27):6466-73.[Abstract][Full Text]
50. Irwin MS, Naranjo A, Zhang FF, et al. Revised Neuroblastoma Risk Classification System: A Report From the Children's Oncology Group. J Clin Oncol. 2021 Oct 10;39(29):3229-41.[Abstract]
51. Nuchtern JG. Perinatal neuroblastoma. Semin Pediatr Surg. 2006 Feb;15(1):10-6.[Abstract]
52. Acharya S, Jayabose S, Kogan SJ, et al. Prenatally diagnosed neuroblastoma. Cancer. 1997 Jul 15;80(2):304-10.[Abstract]
53. Yamamoto K, Hanada R, Kikuchi A, et al. Spontaneous regression of localized neuroblastoma detected by mass screening. J Clin Oncol. 1998 Apr;16(4):1265-9.[Abstract]
54. Oue T, Inoue M, Yoneda A, et al. Profile of neuroblastoma detected by mass screening, resected after observation without treatment: results of the Wait and See pilot study. J Pediatr Surg. 2005 Feb;40(2):359-63.[Abstract]
55. Okazaki T, Kohno S, Mimaya J, et al. Neuroblastoma detected by mass screening: the Tumor Board's role in its treatment. Pediatr Surg Int. 2004 Jan;20(1):27-32.[Abstract]
56. Nishihira H, Toyoda Y, Tanaka Y, et al. Natural course of neuroblastoma detected by mass screening: s 5-year prospective study at a single institution. J Clin Oncol. 2000 Aug;18(16):3012-7.[Abstract]
57. Rubie H, Coze C, Plantaz D, et al. Localised and unresectable neuroblastoma in infants: excellent outcome with low-dose primary chemotherapy. Br J Cancer. 2003 Nov 3;89(9):1605-9.[Abstract][Full Text]
58. 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. 2000 Jan;18(1):18-26.[Abstract]
59. De Bernardi B, Mosseri V, Rubie H, et al. Treatment of localised resectable neuroblastoma. Results of the LNESG1 study by the SIOP Europe Neuroblastoma Group. Br J Cancer. 2008 Oct 7;99(7):1027-33.[Abstract][Full Text]
60. 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. 1997 Mar 5;89(5):373-80.[Abstract][Full Text]
61. Alvarado CS, London WB, Look AT, et al. Natural history and biology of stage A neuroblastoma: a Pediatric Oncology Group Study. J Pediatr Hematol Oncol. 2000 May-Jun;22(3):197-205.[Abstract]
62. Nuchtern JG, London WB, Barnewolt CE, et al. A prospective study of expectant observation as primary therapy for neuroblastoma in young infants: a Children's Oncology Group study. Ann Surg. 2012 Oct;256(4):573-80.[Abstract]
63. Nickerson HJ, Matthay KK, Seeger RC, et al. Favorable biology and outcome of stage IV-S neuroblastoma with supportive care or minimal therapy: a Children's Cancer Group study. J Clin Oncol. 2000 Feb;18(3):477-86.[Abstract]
64. Katzenstein HM, Bowman LC, Brodeur GM, et al. Prognostic significance of age, MYCN oncogene amplification, tumor cell ploidy, and histology in 110 infants with stage D(S) neuroblastoma: the pediatric oncology group experience - a pediatric oncology group study. J Clin Oncol. 1998 Jun;16(6):2007-17.[Abstract]
65. 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. 2008 Mar 20;26(9):1504-10.[Abstract][Full Text]
66. Strother DR, London WB, Schmidt ML, et al. Outcome after surgery alone or with restricted use of chemotherapy for patients with low-risk neuroblastoma: results of Children's Oncology Group study P9641. J Clin Oncol. 2012 May 20;30(15):1842-8.[Abstract][Full Text]
67. Leclair MD, Hartmann O, Heloury Y, et al. Localized pelvic neuroblastoma: excellent survival and low morbidity with tailored therapy - the 10-year experience of the French Society of Pediatric Oncology. J Clin Oncol. 2004 May 1;22(9):1689-95.[Abstract][Full Text]
68. 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. 2000 Mar;18(6):1260-8.[Abstract]
69. 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. 1998 Apr;16(4):1256-64.[Abstract]
70. Bagatell R, Rumcheva P, London WB, et al. Outcomes of children with intermediate-risk neuroblastoma after treatment stratified by MYCN status and tumor cell ploidy. J Clin Oncol. 2005 Dec 1;23(34):8819-27.[Abstract][Full Text]
71. Baker DL, Schmidt ML, Cohn SL, et al. Outcome after reduced chemotherapy for intermediate-risk neuroblastoma. N Engl J Med. 2010 Sep 30;363(14):1313-23.[Abstract][Full Text]
72. Strother D, van Hoff J, Rao PV, et al. Event-free survival of children with biologically favourable neuroblastoma based on the degree of initial tumour resection: results from the Pediatric Oncology Group. Eur J Cancer. 1997 Oct;33(12):2121-5.[Abstract]
73. Strother D, Shuster JJ, McWilliams N, et al. Results of pediatric oncology group protocol 8104 for infants with stages D and DS neuroblastoma. J Pediatr Hematol Oncol. 1995 Aug;17(3):254-9.[Abstract]
74. Mullassery D, Farrelly P, Losty PD. Does aggressive surgical resection improve survival in advanced stage 3 and 4 neuroblastoma? A systematic review and meta-analysis. Pediatr Hematol Oncol. 2014 Nov;31(8):703-16.[Abstract]
75. Zwaveling S, Tytgat GA, van der Zee DC, et al. Is complete surgical resection of stage 4 neuroblastoma a prerequisite for optimal survival or may >95 % tumour resection suffice? Pediatr Surg Int. 2012 Oct;28(10):953-9.[Abstract][Full Text]
76. Yeung F, Chung PH, Tam PK, et al. Is complete resection of high-risk stage IV neuroblastoma associated with better survival? J Pediatr Surg. 2015 Dec;50(12):2107-11.[Abstract]
77. Simon T, Häberle B, Hero B, et al. Role of surgery in the treatment of patients with stage 4 neuroblastoma age 18 months or older at diagnosis. J Clin Oncol. 2013 Feb 20;31(6):752-8.[Abstract][Full Text]
78. McGregor LM, Rao BN, Davidoff AM, et al. The impact of early resection of primary neuroblastoma on the survival of children older than 1 year of age with stage 4 disease: the St. Jude Children's Research Hospital Experience. Cancer. 2005 Dec 15;104(12):2837-46.[Abstract][Full Text]
79. La Quaglia MP, Kushner BH, Heller G, et al. Stage 4 neuroblastoma diagnosed at more than 1 year of age: gross total resection and clinical outcome. J Pediatr Surg. 1994 Aug;29(8):1162-5.[Abstract]
80. Kiely EM. The surgical challenge of neuroblastoma. J Pediatr Surg. 1994 Feb;29(2):128-33.[Abstract]
81. Haase GM, O'Leary MC, Ramsay NK, et al. Aggressive surgery combined with intensive chemotherapy improves survival in poor-risk neuroblastoma. J Pediatr Surg. 1991 Sep;26(9):1119-23.[Abstract]
82. Chamberlain RS, Quinones R, Dinndorf P, et al. Complete surgical resection combined with aggressive adjuvant chemotherapy and bone marrow transplantation prolongs survival in children with advanced neuroblastoma. Ann Surg Oncol. 1995 Mar;2(2):93-100.[Abstract]
83. Bagatell R, McHugh K, Naranjo A, et al. Assessment of primary site response in children with high-risk neuroblastoma: an international multicenter study. J Clin Oncol. 2016 Mar 1;34(7):740-6.[Abstract]
84. Matthay KK, Villablanca JG, Seeger RC, et al. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med. 1999 Oct 14;341(16):1165-73.[Abstract][Full Text]
85. Kreissman SG, Seeger RC, Matthay KK, et al. Purged versus non-purged peripheral blood stem-cell transplantation for high-risk neuroblastoma (COG A3973): a randomised phase 3 trial. Lancet Oncol. 2013 Sep;14(10):999-1008.[Abstract][Full Text]
86. Desai AV, Heneghan MB, Li Y, et al. Toxicities of busulfan/melphalan versus carboplatin/etoposide/melphalan for high-dose chemotherapy with stem cell rescue for high-risk neuroblastoma. Bone Marrow Transplant. 2016 Sep;51(9):1204-10.[Abstract]
87. Proust-Houdemont S, Pasqualini C, Blanchard P, et al. Busulfan-melphalan in high-risk neuroblastoma: the 30-year experience of a single institution. Bone Marrow Transplant. 2016 Aug;51(8):1076-81.[Abstract]
88. Ladenstein R, Pötschger U, Pearson ADJ, et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): an international, randomised, multi-arm, open-label, phase 3 trial. Lancet Oncol. 2017 Apr;18(4):500-14.[Abstract][Full Text]
89. Matthay KK, Reynolds CP, Seeger RC, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a children's oncology group study. J Clin Oncol. 2009 Mar 1;27(7):1007-13.[Abstract][Full Text]
90. Laprie A, Michon J, Hartmann O, et al. High-dose chemotherapy followed by locoregional irradiation improves the outcome of patients with international neuroblastoma staging system stage II and III neuroblastoma with MYCN amplification. Cancer. 2004 Sep 1;101(5):1081-9.[Abstract][Full Text]
91. Mazloom A, Louis CU, Nuchtern J, et al. Radiation therapy to the primary and postinduction chemotherapy MIBG-avid sites in high-risk neuroblastoma. Int J Radiat Oncol Biol Phys. 2014 Nov 15;90(4):858-62.[Abstract]
92. Marcus KJ, Shamberger R, Litman H, et al. Primary tumor control in patients with stage 3/4 unfavorable neuroblastoma treated with tandem double autologous stem cell transplants. J Pediatr Hematol Oncol. 2003 Dec;25(12):934-40.[Abstract]
93. Haas-Kogan DA, Swift PS, Selch M, et al. Impact of radiotherapy for high-risk neuroblastoma: a Children's Cancer Group study. Int J Radiat Oncol Biol Phys. 2003 May 1;56(1):28-39.[Abstract]
94. Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010 Sep 30;363(14):1324-34.[Abstract][Full Text]
95. Ladenstein R, Pötschger U, Gray J, et al. Toxicity and outcome of anti-GD2 antibody ch14.18/CHO in front-line, high-risk patients with neuroblastoma: final results of the phase III immunotherapy randomisation (HR-NBL1/SIOPEN trial). J Clin Oncol. 2016;34(suppl):10500.[Full Text]
96. Garaventa A, Poetschger U, Valteau-Couanet D, et al. Randomized Trial of Two Induction Therapy Regimens for High-Risk Neuroblastoma: HR-NBL1.5 International Society of Pediatric Oncology European Neuroblastoma Group Study. J Clin Oncol. 2021 Aug 10;39(23):2552-63.[Abstract]
97. Peinemann F, van Dalen EC, Enk H, et al. Anti-GD2 antibody-containing immunotherapy postconsolidation therapy for people with high-risk neuroblastoma treated with autologous haematopoietic stem cell transplantation. Cochrane Database Syst Rev. 2019 Apr 24;4:CD012442.[Abstract][Full Text]
98. Peinemann F, van Dalen EC, Enk H, et al. Retinoic acid post consolidation therapy for high-risk neuroblastoma patients treated with autologous hematopoietic stem cell transplantation. Cochrane Database Syst Rev. 2017 Aug 25;8:CD010685.[Abstract][Full Text]
99. Morgenstern DA, Baruchel S, Irwin MS. Current and future strategies for relapsed neuroblastoma: challenges on the road to precision therapy. J Pediatr Hematol Oncol. 2013 Jul;35(5):337-47.[Abstract]
100. Mody R, Naranjo A, Van Ryn C, et al. Irinotecan-temozolomide with temsirolimus or dinutuximab in children with refractory or relapsed neuroblastoma (COG ANBL1221): an open-label, randomised, phase 2 trial. Lancet Oncol. 2017;18(7):946-57.[Abstract][Full Text]
101. Markham A. Naxitamab: First Approval. Drugs. 2021 Feb;81(2):291-96.[Abstract]
102. Zhou MJ, Doral MY, DuBois SG, et al. Different outcomes for relapsed versus refractory neuroblastoma after therapy with (131)I-metaiodobenzylguanidine ((131)I-MIBG). Eur J Cancer. 2015 Nov;51(16):2465-72.[Abstract]
103. Wilson JS, Gains JE, Moroz V, et al. A systematic review of 131I-meta iodobenzylguanidine molecular radiotherapy for neuroblastoma. Eur J Cancer. 2014 Mar;50(4):801-15.[Abstract]
104. George SL, Falzone N, Chittenden S, et al. Individualized 131I-mIBG therapy in the management of refractory and relapsed neuroblastoma. Nucl Med Commun. 2016 May;37(5):466-72.[Abstract]
105. DuBois SG, Granger MM, Groshen S, et al. Randomized phase II trial of MIBG versus MIBG, vincristine, and irinotecan versus MIBG and vorinostat for patients with relapsed or refractory neuroblastoma: a report from NANT consortium. J Clin Oncol. 2021 Nov 1;39(31):3506-14.[Abstract][Full Text]
106. National Cancer Institute. Induction therapy including 131 I-MIBG and chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma undergoing stem cell transplant, radiation therapy, and maintenance therapy with isotretinoin. Jan 2016 [internet publication].[Full Text]
107. DuBois SG, Marachelian A, Fox E, et al. Phase I study of the aurora A kinase inhibitor alisertib in combination with irinotecan and temozolomide for patients with relapsed or refractory neuroblastoma: a NANT (new approaches to neuroblastoma therapy) Trial. J Clin Oncol. 2016 Apr 20;34(12):1368-75.[Abstract][Full Text]
108. Mossé YP, Fox E, Teachey DT, et al. A phase II study of alisertib in children with recurrent/refractory solid tumors or leukemia: children's oncology group phase I and pilot consortium (ADVL0921). Clin Cancer Res. 2019 Jun 1;25(11):3229-38.[Abstract][Full Text]
109. Bassiri H, Benavides A, Haber M, et al. Translational development of difluoromethylornithine (DFMO) for the treatment of neuroblastoma. Transl Pediatr. 2015 Jul;4(3):226-38.[Abstract][Full Text]
110. Saulnier Sholler GL, Gerner EW, Bergendahl G, et al. A phase I trial of DFMO targeting polyamine addiction in patients with relapsed/refractory neuroblastoma. PLoS One. 2015 May 27;10(5):e0127246.[Abstract][Full Text]
111. Sholler GLS, Ferguson W, Bergendahl G, et al. Maintenance DFMO Increases Survival in High Risk Neuroblastoma. Sci Rep. 2018 Sep 27;8(1):14445.[Abstract][Full Text]
112. Louis CU, Savoldo B, Dotti G, et al. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood. 2011 Dec 1;118(23):6050-6.[Abstract][Full Text]
113. ClinicalTrials.gov. 131I-burtomab radioimmunotherapy for neuroblastoma central nervous system/leptomeningeal metastases. ClinicalTrials.gov identifier: NCT03275402. Dec 2021 [internet publication].[Full Text]
114. ClinicalTrials.gov. Enoblituzumab (MGA271) in children with B7-H3-expressing solid tumors. ClinicalTrials.gov identifier: NCT02982941. Feb 2022 [internet publication].[Full Text]
115. ClinicalTrials.gov. Testing the combination of two immunotherapy drugs (magrolimab and dinutuximab) in patients with relapsed or refractory neuroblastoma or relapsed osteosarcoma. ClinicalTrials.gov identifier: NCT04751383. Mar 2022 [internet publication].[Full Text]
116. ClinicalTrials.gov. C7R-GD2.CART cells for patients with relapsed or refractory neuroblastoma and other GD2 positive cancers (GAIL-N). ClinicalTrials.gov identifier: NCT03635632. Nov 2021 [internet publication].[Full Text]
117. ClinicalTrials.gov. GD2 specific CAR and interleukin-15 expressing autologous NKT cells to treat children with neuroblastoma (GINAKIT2). ClinicalTrials.gov identifier: NCT03294954. Jan 2022 [internet publication].[Full Text]
118. ClinicalTrials.gov. Study of the safety and efficacy of humanized 3F8 bispecific antibody (Hu3F8-BsAb) in patients with relapsed/refractory neuroblastoma, osteosarcoma and other solid tumor cancers. ClinicalTrials.gov identifier: NCT03860207. May 2021 [internet publication].[Full Text]
119. National Cancer Institute. Neuroblastoma treatment - health professional version (PDQ). February 2022 [Internet publication].[Full Text]
120. Sokol E, Desai AV, Applebaum MA, et al. Age, Diagnostic Category, Tumor Grade, and Mitosis-Karyorrhexis Index Are Independently Prognostic in Neuroblastoma: An INRG Project. J Clin Oncol. 2020 Jun 10;38(17):1906-18.[Abstract][Full Text]
121. Yalçin B, Kremer LC, van Dalen EC. High-dose chemotherapy and autologous haematopoietic stem cell rescue for children with high-risk neuroblastoma. Cochrane Database Syst Rev. 2015;(10):CD006301.[Abstract][Full Text]
122. Simon T, Berthold F, Borkhardt A, et al. Treatment and outcomes of patients with relapsed, high-risk neuroblastoma: results of German trials. Pediatr Blood Cancer. 2011 Apr;56(4):578-83.[Abstract]
123. Santana VM, Furman WL, McGregor LM, et al. Disease control intervals in high-risk neuroblastoma. Cancer. 2008 Jun 15;112(12):2796-801.[Abstract][Full Text]
124. Cole KA, Maris JM. New strategies in refractory and recurrent neuroblastoma: translational opportunities to impact patient outcome. Clin Cancer Res. 2012 May 1;18(9):2423-8.[Abstract][Full Text]
125. Garaventa A, Parodi S, De Bernardi B, et al. Outcome of children with neuroblastoma after progression or relapse. A retrospective study of the Italian neuroblastoma registry. Eur J Cancer. 2009 Nov;45(16):2835-42.[Abstract]
126. Moreno L, Vaidya SJ, Pinkerton CR, et al. Long-term follow-up of children with high-risk neuroblastoma: the ENSG5 trial experience. Pediatr Blood Cancer. 2013 Jul;60(7):1135-40.[Abstract]
127. Landier W, Knight K, Wong FL, et al. Ototoxicity in children with high-risk neuroblastoma: prevalence, risk factors, and concordance of grading scales - a report from the Children's Oncology Group. J Clin Oncol. 2014 Feb 20;32(6):527-34.[Abstract][Full Text]
128. Hobbie WL, Moshang T, Carlson CA, et al. Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma. Pediatr Blood Cancer. 2008 Nov;51(5):679-83.[Abstract][Full Text]
129. Grewal S, Merchant T, Reymond R, et al. Auditory late effects of childhood cancer therapy: a report from the Children's Oncology Group. Pediatrics. 2010 Apr;125(4):e938-50.[Abstract][Full Text]
130. French AE, Irwin MS, Navarro OM, et al. Long-term hepatic outcomes in survivors of stage 4S and 4 neuroblastoma in infancy. Pediatr Blood Cancer. 2012 Feb;58(2):283-8.[Abstract]
131. van Casteren NJ, van der Linden GH, Hakvoort-Cammel FG, et al. Effect of childhood cancer treatment on fertility markers in adult male long-term survivors. Pediatr Blood Cancer. 2009 Jan;52(1):108-12.[Abstract]
132. Ducassou A, Gambart M, Munzer C, et al. Long-term side effects of radiotherapy for pediatric localized neuroblastoma : results from clinical trials NB90 and NB94. Strahlenther Onkol. 2015 Jul;191(7):604-12.[Abstract]
133. Kelly J, Damron T, Grant W, et al. Cross-sectional study of bone mineral density in adult survivors of solid pediatric cancers. J Pediatr Hematol Oncol. 2005 May;27(5):248-53.[Abstract]
134. Simon T, Niemann CA, Hero B, et al. Short- and long-term outcome of patients with symptoms of spinal cord compression by neuroblastoma. Dev Med Child Neurol. 2012 Apr;54(4):347-52.[Abstract][Full Text]
135. Cohen LE, Gordon JH, Popovsky EY, et al. Late effects in children treated with intensive multimodal therapy for high-risk neuroblastoma: high incidence of endocrine and growth problems. Bone Marrow Transplant. 2014 Apr;49(4):502-8.[Abstract][Full Text]
136. Rudnick E, Khakoo Y, Antunes NL, et al. Opsoclonus-myoclonus-ataxia syndrome in neuroblastoma: clinical outcome and antineuronal antibodies-a report from the Children's Cancer Group Study. Med Pediatr Oncol. 2001 Jun;36(6):612-22.[Abstract]
137. Applebaum MA, Henderson TO, Lee SM, et al. Second malignancies in patients with neuroblastoma: the effects of risk-based therapy. Pediatr Blood Cancer. 2015 Jan;62(1):128-33.[Abstract][Full Text]
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