Neuroblastoma: Contemporary management

Archives of Disease in Childhood: Education and Practice Edition

Volumn 94, Issue 6, 2009, Pages 177-185

  Mullassery, D ^a   Dominici, C ^a,b,c   Jesudason, C ^a   McDowell, H P ^b,d   Losty, P D ^a  

^a UNIVERSITY OF LIVERPOOL   (United Kingdom)

^b SAPIENZA UNIVERSITY OF ROME   (Italy)

^c BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^d ALDER HEY CHILDREN'S NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTI GANGLIOSIDE D2 ANTIBODY; CARBOPLATIN; CISPLATIN; CYCLOPHOSPHAMIDE; DOXORUBICIN; ETOPOSIDE; GANGLIOSIDE ANTIBODY; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY 3F8; RETINOIC ACID; TOPOTECAN; UNCLASSIFIED DRUG; VINCRISTINE;

CANCER RELAPSE; CANCER STEM CELL; CANCER SURGERY; CLINICAL FEATURE; CLINICAL TRIAL; DRUG MEGADOSE; EXTERNAL BEAM RADIOTHERAPY; GENETIC PREDISPOSITION; HUMAN; MINIMAL RESIDUAL DISEASE; NEUROBLASTOMA; PRIORITY JOURNAL; PROGNOSIS; REVIEW; RISK FACTOR; URINALYSIS;

ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CHILD; DIAGNOSTIC IMAGING; GENETIC PREDISPOSITION TO DISEASE; HUMANS; NEOPLASM REGRESSION, SPONTANEOUS; NEOPLASM STAGING; NEOPLASM, RESIDUAL; NEUROBLASTOMA; PRACTICE GUIDELINES AS TOPIC; PROGNOSIS; RISK FACTORS; TUMOR MARKERS, BIOLOGICAL;

EID: 73649095554     PISSN: 17430585     EISSN: 17430593     Source Type: Journal    
DOI: 10.1136/adc.2008.143909     Document Type: Review

References (93)

1. Ries LAG, Smith MA, Gurney JG, et al. Cancer incidence and survival among children and adolescents: United States SEER program 1975-1995. NIH publication no. 99-4649. Bethesda, Maryland, USA: National Cancer Institute, SEER Program, 1999.
2. De Bernardi B, Nicolas B, Boni L, et al. Disseminated neuroblastoma in children older than one year at diagnosis: comparable results with three consecutive high-dose protocols adopted by the Italian co-operative group for neuroblastoma. J Clin Oncol 2003;21:1592-601.
3. Maris JM, Hogarty MD, Bagatell R, et al. Neuroblastoma. Lancet 2007;369:2106-20.
4. Stiller CA, Parkin DM. International variations in the incidence of neuroblastoma. Int J Cancer 1992;52:538-43.
5. Shojaei-Brosseau T, Chompret A, Abel A, et al. Genetic epidemiology of neuroblastoma: a study of 426 cases at the Institut Gustave-Roussy in France. Pediatr Blood Cancer 2004;42:99-105.
6. Rohrer T, Trachsel D, Engelcke G, et al. Congenital central hypoventilation syndrome associated with Hirschsprung's disease and neuroblastoma: case of multiple neurocristopathies. Pediatr Pulmonol 2002;33:71-6.
7. Gaultier C, Trang H, Dauger S, et al. Pediatric disorderswith autonomic dysfunction: what role for PHOX2B? Pediatr Res 2005;58:1-6.
8. Kushner BH, Hajdu SI, Helson L. Synchronous neuroblastoma and von Recklinghausen's disease: a review of the literature. J Clin Oncol 1985;3:117-20.
9. Dick JE. Stem cell concepts renew cancer research. Blood 2008;112:4793-807.
10. Ross RA, Spengler BA. Human neuroblastoma stem cells. Semin Cancer Biol 2007;17:241-7.
11. Shimada H, Umehara S, Monobe Y, et al. International neuroblastoma pathology classification for prognostic evaluation of patients with peripheral neuroblastic tumors: a report from the Children's Cancer Group. Cancer 2001;92:2451-61.
12. Seeger RC, Brodeur GM, Sather H, et al. Association of multiple copies of the N-myc oncogene with rapid progression of neuroblastomas. N Engl J Med 1985;313:1111-6.
13. Maris JM, Weiss MJ, Guo C, et al. Loss of heterozygosity at 1p36 independently predicts for disease progression but not decreased overall survival probability in neuroblastoma patients: a Children's Cancer Group study. J Clin Oncol 2000;18:1888-99.
14. Attiyeh EF, London WB, Mosse YP, et al. Chromosome 1p and 11q deletions and outcome in neuroblastoma. N Engl J Med 2005;353:2243-53.
15. Bown N, Cotterill S, Lastowska M, et al. Gain of chromosome arm 17q and adverse outcome in patients with neuroblastoma. N Engl J Med 1999;340:1954-61.
16. Look AT, Hayes FA, Nitschke R, et al. Cellular DNA content as a predictor of response to chemotherapy in infants with unresectable neuroblastoma. N Engl J Med 1984;311:231-5.
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 2005;23:6466-73.
18. Ambros PF, Ambros IM, Brodeur GM, et al. International consensus for neuroblastoma molecular diagnostics: report from the International Neuroblastoma Risk Group (INRG) biology committee. Br J Cancer 2009;100:1471-82.
19. Wang Q, Diskin S, Rappaport E, et al. Integrative genomics identifies distinct molecular classes of neuroblastoma and shows that multiple genes are targeted by regional alterations in DNA copy number. Cancer Res 2006;66:6050-62.
20. Lastowska M, Viprey V, Santibanez-Koref M, et al. Identification of candidate genes involved in neuroblastoma progression by combining genomic and expression microarrays with survival data. Oncogene 2007;26:7432-44.
21. Mosse YP, Diskin SJ, Wasserman N, et al. Neuroblastomas have distinct genomic DNA profiles that predict clinical phenotype and regional gene expression. Genes Chromosomes Cancer 2007;46:936-49.
22. Hann HL, Evans AE, Siegel SE, et al. Prognostic importance of serum ferritin in patients with stages III and IV neuroblastoma: the Childrens Cancer Study Group experience. Cancer Res 1985;45:2843- 8.
23. Zeltzer PM, Marangos PJ, Evans AE, et al. Serum neuron-specific enolase in children with neuroblastoma: relationship to stage and disease course. Cancer 1986;57:1230-4.
24. Joshi VV, Cantor AB, Brodeur GM, et al. Correlation between morphologic and other prognostic markers of neuroblastoma: a study of histologic grade, DNA index, N-myc gene copy number, and lactic dehydrogenase in patients in the Pediatric Oncology Group. Cancer 1993;71:3173-81.
25. Hsiao RJ, seeger RC, Yu AL, et al. Chromogranin A in children with neuroblastoma: serum concentrations parallels disease stage and predicts survival. J Clin Invest 1990;85:1555-9.
26. Strenger V, Kerbl R, Dornbusch HJ, et al. Diagnostic and prognostic impact of urinary catecholamines in neuroblastoma patients. Pediatr Blood Cancer 2007;48:504-9.
27. Kaste SC, McCarville MB. Imaging pediatric abdominal tumors. Semin Roentgenol 2008;43:50-9.
28. McHugh K, Pritchard J. Problems in the imaging of three common paediatric solid tumours. Eur J Radiol 2001;37:72-8.
29. Gelfand MJ. Meta-iodobenzylguanidine in children. Semin Nucl Med 1993;23:231-42.
30. Kushner BH. Neuroblastoma: a disease requiring a multitude of imaging studies. J Nucl Med 2004;45:1172-88.
31. Shulkin BL, Hutchinson RJ, Castle VP, et al. Neuroblastoma: positron emission tomography with 2-[fluorine-18]-fluoro-2-deoxy-D- glucose compared with metaiodobenzylguanidine scintigraphy. Radiology 1996;199:743-50.
32. Dowell HM, Losty P, Barnes N, et al. Utility of FDG-PET/CT in the follow-up of neuroblastoma which became MIBG-negative. Pediatr Blood Cancer 2008;52:552.
33. Gupta A, Kumar A, Walters S, et al. Analysis of needle versus open biopsy for the diagnosis of advanced stage pediatric neuroblastoma. Pediatr Blood Cancer 2006;47:875-9.
34. Iwanaka T, Kawashima H, Uchida H. The laparoscopic approach of neuroblastoma. Semin Pediatr Surg 2007;16:259-65.
35. 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;11:1466-77.
36. Cohn SL, Pearson AD, London WB, et al. The International Neuroblastoma Risk Group (INRG) classification system: an INRG Task Force report. J Clin Oncol 2009;27:289-97.
37. Cecchetto G, Mosseri V, De Bernardi B, et al. Surgical risk factors in primary surgery for localized neuroblastoma: the LNESG1 study of the European International Society of Pediatric Oncology Neuroblastoma Group. J Clin Oncol 2005;23:8483-9.
38. Simon T, Hero B, Benz-Bohm G, et al. Review of image defined risk factors in localized neuroblastoma patients: results of the GPOH NB97 trial. Pediatr Blood Cancer 2008;50:965-9.
39. Evans AE, Gerson J, Schnaufer L. Spontaneous regression of neuroblastoma. Natl Cancer Inst Monogr 1976;44:49-54.
40. Brodeur GM. Neuroblastoma: biological insights into a clinical enigma. Nat Rev Cancer 2003;3:203-16.
41. D'Angio GJ, Evans AE, Koop CE. Special pattern of widespread neuroblastoma with a favourable prognosis. Lancet 1971;1:1046-9.
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;20:1209-14.
43. Woods WG, Gao RN, Shuster JJ, et al. Screening of infants and mortality due to neuroblastoma. N Engl J Med 2002;346:1041-6.
44. Schilling FH, Spix C, Berthold F, et al. Neuroblastoma screening at one year of age. N Engl J Med 2002;346:1047-53.
45. Kubota M, Yagi M, Kanada S, et al. Long-term follow-up status of patients with neuroblastoma after undergoing either aggressive surgery or chemotherapy: a single institutional study. J Pediatr Surg 2004;39:1328-32.
46. Kiely EM. The surgical challenge of neuroblastoma. J Pediatr Surg 1994;29:128-33.
47. Kiely E. A technique for excision of abdominal and pelvic neuroblastomas. Ann R Coll Surg Engl 2007;89:342-8.
48. Losty P, Quinn F, Breatnach F, et al. Neuroblastoma: a surgical perspective. Eur J Surg Oncol 1993;19:33-6.
49. Kushner BH, LaQuaglia MP, Kramer K, et al. Radically different treatment recommendations for newly diagnosed neuroblastoma: pitfalls in assessment of risk. J Pediatr Hematol Oncol 2004;26:35-9.
50. Leclair MD, de Lagausie P, Becmeur F, et al. Laparoscopic resection of abdominal neuroblastoma. Ann Surg Oncol 2008;15:117- 24.
51. Adkins ES, Sawin R, Gerbing RB, et al. Efficacy of complete resection for high-risk neuroblastoma: a Children's Cancer Group study. J Pediatr Surg 2004;39:931-6.
52. 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;26:1504-10.
53. Pinkerton CR, Blanc Vincent MP, Bergeron C, et al. Induction chemotherapy in metastatic neuroblastoma: does dose influence response? A critical review of published data standards, options and recommendations (SOR) project of the National Federation of French Cancer Centres (FNCLCC). Eur J Cancer 2000;36:1808-15.
54. Pearson AD, Pinkerton CR, Lewis IJ, et al. High-dose rapid and standard induction chemotherapy for patients aged over 1 year with stage 4 neuroblastoma: a randomised trial. Lancet Oncol 2008;9:247-56.
55. Saylors RL 3rd, Stine KC, Sullivan J, et al. Cyclophosphamide plus topotecan in children with recurrent or refractory solid tumors: a Pediatric Oncology Group phase II study. J Clin Oncol 2001;19:3463-9.
56. Kretschmar CS, Kletzel M, Murray K, et al. Response to paclitaxel, topotecan, and topotecan-cyclophosphamide in children with untreated disseminated neuroblastoma treated in an upfront phase II investigational window: a Pediatric Oncology Group study. J Clin Oncol 2004;22:4119-26.
57. Park JR, London WB, Santana VM, et al. A topotecan-containing induction regimen for treatment of high-risk neuroblastoma. J Clin Oncol 2006;24:9013.
58. Matthay KK, Villablanca JG, Seeger RC, et al. Treatment of highrisk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. N Engl J Med 1999;341:1165-73.
59. 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;27:1007-13.
60. Pritchard J, Cotterill SJ, Germond SM, et al. High dose melphalan in the treatment of advanced neuroblastoma: results of a randomised trial (ENSG-1) by the European Neuroblastoma Study Group. Pediatr Blood Cancer 2005;44:348-57.
61. Berthold F, Boos J, Burdach S, et al. Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial. Lancet Oncol 2005;6:649-58.
62. Fish JD, Grupp SA. Stem cell transplantation for neuroblastoma. Bone Marrow Transplant 2008;41:159-65.
63. George RE, Li S, Medeiros-Nancarrow C, et al. High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: long-term survival update. J Clin Oncol 2006;24:2891-6.
64. Cheung NK, Kushner BH, LaQuaglia M, et al. N7: a novel multimodality therapy of high risk neuroblastoma (NB) in children diagnosed over 1 year of age. Med Pediatr Oncol 2001;36:227-30.
65. Kushner BH, Kramer K, LaQuaglia MP, et al. Reduction from seven to five cycles of intensive induction chemotherapy in children with high-risk neuroblastoma. J Clin Oncol 2004;22:4888-92.
66. De Bernardi B, Pianca C, Pistamiglio P, et al. Neuroblastoma with symptomatic spinal cord compression at diagnosis: treatment and results with 76 cases. J Clin Oncol 2001;19:183-90.
67. Reynolds CP. Detection and treatment of minimal residual disease in high-risk neuroblastoma. Pediatr Transplant 2004;8(Suppl 5):56-66.
68. Beiske K, Burchill SA, Cheung IK, et al. Consensus criteria for sensitive detection of minimal neuroblastoma cells in bone marrow, blood and stem cells preparations by immunocytology and QRT-PCR: recommendations by the International Neuroblastoma Risk Group Task Force. Br J Cancer 2009;100:1627-37.
69. Reynolds CP, Matthay KK, Villablanca JG, et al. Retinoid therapy of high-risk neuroblastoma. Cancer Lett 2003;197:185-92.
70. Villablanca JG, Krailo MD, Ames MM, et al. Phase I trial of oral fenretinide in children with high-risk solid tumors: a report from the Children's Oncology Group (CCG 09709). J Clin Oncol 2006;24:3423- 30.
71. Modak S, Cheung NK. Disialoganglioside directed immunotherapy of neuroblastoma. Cancer Invest 2007;25:67-77.
72. Ozkaynak MF, Sondel PM, Krailo MD, et al. Phase I study of chimeric human/murine anti-ganglioside GD2 monoclonal antibody (ch14.18) with granulocyte-macrophage colony-stimulating factor in children with neuroblastoma immediately after hematopoietic stem-cell transplantation: a Children's Cancer Group Study. J Clin Oncol 2000;18:4077-85.
73. D2 monoclonal antibody 3F8 and granulocyte-macrophage colony-stimulating factor for neuroblastoma. J Clin Oncol 2001;19:4189-94.
74. Yu AL, Gilman AL, Ozkaynak MF, et al. A phase III randomized trial of the chimeric anti-GD2 antibody ch14.18 with GM-CSF and IL2 as immunotherapy following dose intensive chemotherapy for high-risk neuroblastoma: Children's Oncology Group (COG) study ANBL0032. J Clin Oncol 2009;27(Suppl):15s.
75. Stempak D, Seely D, Baruchel S. Metronomic dosing of chemotherapy: applications in pediatric oncology. Cancer Invest 2006;24:432-43.
76. Garaventa A, Luksch R, Biasotti S, et al. A phase II study of topotecan with vincristine and doxorubicin in children with recurrent/refractory neuroblastoma. Cancer 2003;98:2488-94.
77. Kusner BH, Kramer K, Modak S, et al. Irinotecan plus temozolomide for relapsed or refractory neuroblastoma. J Clin Oncolo 2006;24:5271-6.
78. Matthay KK, Yanik G, Messina J, et al. Phase II study on the effect of disease sites, age, and prior therapy on response to iodine-131-metaiodobenzylguanidine therapy in refractory neuroblastoma. J Clin Oncol 2007;25:1054-60.
79. Matthay KK, Quach A, Huberty J, et al. Iodine-131- metaiodobenzylguanidine double infusion with autologous stem-cell rescue for neuroblastoma: a new approaches to neuroblastoma therapy phase I study. J Clin Oncol 2009;27:1020-5.
80. Laverdière C, Liu Q, Yasui Y, et al. Long-term outcomes in survivors of neuroblastoma: a report from the Childhood Cancer Survivor Study. J Natl Cancer Inst 2009;101:1131-40.
81. Evans AE, Kisselbach KD, Liu X, et al. Effect of CEP-751 (KT-6587) on neuroblastoma xenografts expressing TrkB. Med Pediatr Oncol 2001;36:181-4.
82. Vitali R, Cesi V, Nicotra MR, et al. c-Kit is preferentially expressed in MYCN-amplified neuroblastoma and its effect on cell proliferation is inhibited in vitro by STI-571. Int J Cancer 2003;106:147-52.
83. Ho R, Minturn JE, Hishiki T, et al. Proliferation of human neuroblastomas mediated by the epidermal growth factor receptor. Cancer Res 2005;65:9868-75.
84. Tanno B, Mancini C, Vitali R, et al. Down-regulation of insulin-like growth factor I receptor activity by NVP-AEW541 has an antitumor effect on neuroblastoma cells in vitro and in vivo. Clin Cancer Res 2006;12:6772-80.
85. Maris JM, Courtright J, Houghton PJ, et al. Initial testing of the VEGFR inhibitor AZD2171 by the pediatric preclinical testing program. Pediatr Blood Cancer 2008;50:581-7.
86. George RE, Sanda T, Hanna M, et al. Activating mutations in ALK provide a therapeutic target in neuroblastoma. Nature 2008;455:975-8.
87. Brignole C, Marimpietri D, Pastorino F, et al. Effect of bortezomib on human neuroblastoma cell growth, apoptosis, and angiogenesis. J Natl Cancer Inst 2006;98:1142-57.
88. Van Maerken T, Speleman F, Vermeulen J, et al. Small-molecule MDM2 antagonists as a new therapy concept for neuroblastoma. Cancer Res 2006;66:9646-55.
89. Segerström L, Fuchs D, Bäckman U, et al. The anti-VEGF antibody bevacizumab potently reduces the growth rate of high-risk neuroblastoma xenografts. Pediatr Res 2006;60:576-81.
90. Johnsen JI, Segerström L, Orrego A, et al. Inhibitors of mammalian target of rapamycin downregulate MYCN protein expression and inhibit neuroblastoma growth in vitro and in vivo. Oncogene 2008;27:2910-22.
91. Ponthan F, Wickström M, Gleissman H, et al. Celecoxib prevents neuroblastoma tumor development and potentiates the effect of chemotherapeutic drugs in vitro and in vivo. Clin Cancer Res 2007;13:1036-44.
92. Palmberg E, Johnsen JI, Paulsson J, et al. Metronomic scheduling of imatinib abrogates clonogenicity of neuroblastoma cells and enhances their susceptibility to selected chemotherapeutic drugs in vitro and in vivo. Int J Cancer 2009;124:1227-34.
93. Donfrancesco A, De Ioris MA, McDowell HP, et al. Gefitinib in combination with oral topotecan and cyclophosphamide in resistant/relapsed neuroblastoma: pharmacological rationale and clinical response. Pediatr Blood Cancer 2009. In press.