Current and future strategies for relapsed neuroblastoma: Challenges on the road to precision therapy

Journal of Pediatric Hematology/Oncology

Volumn 35, Issue 5, 2013, Pages 337-347

  Morgenstern, Daniel A ^a   Baruchel, Sylvain ^a   Irwin, Meredith S ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

131I MIBG; Immunotherapy; Irinotecan; Neuroblastoma; Precision medicine; Topotecan

Indexed keywords

(3 IODOBENZYL)GUANIDINE I 131; 1 CYCLOPROPYL 3 [3 (5 MORPHOLINOMETHYL 1H BENZIMIDAZOL 2 YL) 1H PYRAZOL 4 YL]UREA; ALISERTIB; ANTINEOPLASTIC AGENT; BEVACIZUMAB; CAMPTOTHECIN; CARBOPLATIN; CRIZOTINIB; CYCLOPHOSPHAMIDE; DOXORUBICIN; ETOPOSIDE; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; IFOSFAMIDE; IMATINIB; INTERLEUKIN 2; IRINOTECAN; LDK 378; LENALIDOMIDE; LESTAURTINIB; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY CH14.18; PERIFOSINE; PROTEIN TYROSINE KINASE INHIBITOR; TEMOZOLOMIDE; TEMSIROLIMUS; TOPOTECAN; UNCLASSIFIED DRUG; UNINDEXED DRUG; VINCRISTINE; VORINOSTAT; ZOLEDRONIC ACID;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; BACTEREMIA; BONE MARROW SUPPRESSION; BONE MARROW TOXICITY; CANCER COMBINATION CHEMOTHERAPY; CANCER IMMUNOTHERAPY; CANCER RADIOTHERAPY; CANCER SURVIVAL; CANCER THERAPY; CAPILLARY LEAK SYNDROME; CENTRAL NERVOUS SYSTEM METASTASIS; CLINICAL EFFECTIVENESS; CONTINUOUS INFUSION; DIARRHEA; DRUG EFFECT; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG MEGADOSE; DRUG POTENCY; DRUG POTENTIATION; DRUG SAFETY; DRUG TOLERABILITY; EXTERNAL BEAM RADIOTHERAPY; HUMAN; HYPOTENSION; HYPOTHYROIDISM; NEUROBLASTOMA; NEUTROPENIA; NONHUMAN; PAIN; PRIORITY JOURNAL; RADIATION DOSE FRACTIONATION; RECURRENT CANCER; REVIEW; SALVAGE THERAPY; SOLID TUMOR; THROMBOCYTOPENIA; TREATMENT DURATION; TREATMENT PLANNING;

ANTINEOPLASTIC AGENTS; HUMANS; IMMUNOTHERAPY; MEDICAL ONCOLOGY; NEOPLASM RECURRENCE, LOCAL; NEUROBLASTOMA; RADIOTHERAPY;

EID: 84880313396     PISSN: 10774114     EISSN: 15363678     Source Type: Journal    
DOI: 10.1097/MPH.0b013e318299d637     Document Type: Review

References (144)

1. Smith MA, Seibel NL, Altekruse SF, et al. Outcomes for children and adolescents with cancer: challenges for the twenty-first century. J Clin Oncol. 2010;28:2625-2634.
2. 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;27:1007-1013.
3. Cole KA, Maris JM. New strategies in refractory and recurrent neuroblastoma: translational opportunities to impact patient outcome. Clin Cancer Res. 2012;18:2423-2428.
4. Santana VM, Furman WL, Mcgregor LM, et al. Disease control intervals in high-risk neuroblastoma. Cancer. 2008; 112:2796-2801.
5. 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;56: 578-583.
6. 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;45:2835-2842.
7. London WB, Castel V, Monclair T, et al. Clinical and biologic features predictive of survival after relapse of neuroblastoma: a report from the International Neuroblastoma Risk Group project. J Clin Oncol. 2011;29: 3286-3292.
8. Lau L, Tai D, Weitzman S, et al. Factors influencing survival in children with recurrent neuroblastoma. J Pediatr Hematol Oncol. 2004;26:227-232.
9. Castel V, Villamo n E, Can ete A, et al. Neuroblastoma in adolescents: genetic and clinical characterisation. Clin Transl Oncol. 2010;12:49-54.
10. Conte M, Parodi S, De Bernardi B, et al. Neuroblastoma in adolescents: the Italian experience. Cancer. 2006;106: 1409-1417.
11. Franks LM, Bollen A, Seeger RC, et al. Neuroblastoma in adults and adolescents: an indolent course with poor survival. Cancer. 1997;79:2028-2035.
12. Blatt J, Gula MJ, Orlando SJ, et al. Indolent course of advanced neuroblastoma in children older than 6 years at diagnosis. Cancer. 1995;76:890-894.
13. Gaspar N, Hartmann O, Munzer C, et al. Neuroblastoma in adolescents. Cancer. 2003;98:349-355.
14. Cheung N-KV, Zhang J, Lu C, et al. Association of age at diagnosis and genetic mutations in patients with neuroblastoma. JAMA. 2012;307:1062-1071.
15. Castelo-Branco P, Zhang C, Lipman T, et al. Neural tumorinitiating cells have distinct telomere maintenance and can be safely targeted for telomerase inhibition. Clin Cancer Res. 2011;17:111-121.
16. Bagatell R, London WB, Wagner LM, et al. Phase II study of irinotecan and temozolomide in children with relapsed or refractory neuroblastoma: a Children's Oncology Group study. J Clin Oncol. 2011;29:208-213.
17. Shusterman S, London WB, Gillies SD, et al. Antitumor activity of hu14.18-IL2 in patients with relapsed/refractory neuroblastoma: a Children's Oncology Group (COG) phase II study. J Clin Oncol. 2010;28:4969-4975.
18. Beiske K, Ambros PF, Burchill SA, et al. Detecting minimal residual disease in neuroblastoma patients-the present state of the art. Cancer Lett. 2005;228:229-240.
19. Stutterheim J, Gerritsen A, Zappeij-Kannegieter L, et al. Detecting minimal residual disease in neuroblastoma: the superiority of a panel of real-time quantitative PCR markers. Clin Chem. 2009;55:1316-1326.
20. 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;11:1466-1477.
21. Brodeur GM, Seeger RC, Barrett A, et al. International criteria for diagnosis, staging, and response to treatment in patients with neuroblastoma. J Clin Oncol. 1988;6:1874-1881.
22. Simon T, La ngler A, Harnischmacher U, et al. Topotecan, cyclophosphamide, and etoposide (TCE) in the treatment of high-risk neuroblastoma. Results of a phase-II trial. J Cancer Res Clin Oncol. 2007;133:653-661.
23. London WB, Frantz CN, Campbell LA, et al. Phase II randomized comparison of topotecan plus cyclophosphamide versus topotecan alone in children with recurrent or refractory neuroblastoma: a Children's Oncology Group study. J Clin Oncol. 2010;28:3808-3815.
24. Park JR, Scott JR, Stewart CF, et al. Pilot induction regimen incorporating pharmacokinetically guided topotecan for treatment of newly diagnosed high-risk neuroblastoma: a Children's Oncology Group study. J Clin Oncol. 2011;29: 4351-4357.
25. Zamboni WC, Stewart CF, Thompson J, et al. Relationship between topotecan systemic exposure and tumor response in human neuroblastoma xenografts. J Natl Cancer Inst. 1998; 90:505-511.
26. Houghton PJ, Cheshire PJ, Hallman JD, et al. Efficacy of topoisomerase I inhibitors, topotecan and irinotecan, administered at low dose levels in protracted schedules to mice bearing xenografts of human tumors. Cancer Chemother Pharmacol. 1995;36:393-403.
27. Vassal G, Terrier-Lacombe MJ, Bissery MC, et al. Therapeutic activity of CPT-11, a DNA-topoisomerase I inhibitor, against peripheral primitive neuroectodermal tumour and neuroblastoma xenografts. Br J Cancer. 1996;74:537-545.
28. Santos A, Calvet L, Terrier-Lacombe M-J, et al. In vivo treatment with CPT-11 leads to differentiation of neuroblastoma xenografts and topoisomerase I alterations. Cancer Res. 2004;64:3223-3229.
29. Carol H, Houghton PJ, Morton CL, et al. Initial testing of topotecan by the pediatric preclinical testing program. Pediatr Blood Cancer. 2010;54:707-715.
30. Komuro H, Li P, Tsuchida Y, et al. Effects of CPT-11 (a unique DNA topoisomerase I inhibitor) on a highly malignant xeno-transplanted neuroblastoma. Med Pediatr Oncol. 1994;23:487-492.
31. Thompson J, Zamboni WC, Cheshire PJ, et al. Efficacy of oral irinotecan against neuroblastoma xenografts. Anticancer Drugs. 1997;8:313-322.
32. Thompson J, Zamboni WC, Cheshire PJ, et al. Efficacy of systemic administration of irinotecan against neuroblastoma xenografts. Clin Cancer Res. 1997;3:423-431.
33. Blaney S, Berg SL, Pratt C, et al. A phase I study of irinotecan in pediatric patients: a pediatric oncology group study. Clin Cancer Res. 2001;7:32-37.
34. Furman WL, Stewart CF, Poquette CA, et al. Direct translation of a protracted irinotecan schedule from a xenograft model to a phase I trial in children. J Clin Oncol. 1999;17:1815-1824.
35. Blaney SM, Balis FM, Cole DE, et al. Pediatric phase I trial and pharmacokinetic study of topotecan administered as a 24-hour continuous infusion. Cancer Res. 1993;53: 1032-1036.
36. Baker SD, Heideman RL, Crom WR, et al. Cerebrospinal fluid pharmacokinetics and penetration of continuous infusion topotecan in children with central nervous system tumors. Cancer Chemother Pharmacol. 1996;37:195-202.
37. Blaney SM, Needle MN, Gillespie A, et al. Phase II trial of topotecan administered as 72-hour continuous infusion in children with refractory solid tumors: a collaborative Pediatric Branch, National Cancer Institute, and Children's Cancer Group Study. Clin Cancer Res. 1998;4:357-360.
38. Hawkins DS, Bradfield S, Whitlock JA, et al. Topotecan by 21-day continuous infusion in children with relapsed or refractory solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2006;47:790-794.
39. La ngler A, Christaras A, Abshagen K, et al. Topotecan in the treatment of refractory neuroblastoma and other malignant tumors in childhood-a phase-II-study. Klin Padiatr. 2002; 214:153-156.
40. Nitschke R, Parkhurst J, Sullivan J, et al. Topotecan in pediatric patients with recurrent and progressive solid tumors: a Pediatric Oncology Group phase II study. J Pediatr Hematol Oncol. 1998;20:315-318.
41. Bomgaars LR, Bernstein M, Krailo M, et al. Phase II trial of irinotecan in children with refractory solid tumors: a Children's Oncology Group Study. J Clin Oncol. 2007;25: 4622-4627.
42. Shitara T, Shimada A, Hanada R, et al. Irinotecan for children with relapsed solid tumors. Pediatr Hematol Oncol. 2006;23:103-110.
43. Vassal G, Giammarile F, Brooks M, et al. A phase II study of irinotecan in children with relapsed or refractory neuroblastoma: a European cooperation of the Societe Francaise d'Oncologie Pediatrique (SFOP) and the United Kingdom Children Cancer Study Group (UKCCSG). Eur J Cancer. 2008;44:2453-2460.
44. Rodriguez-Galindo C, Crews KR, Stewart CF, et al. Phase I study of the combination of topotecan and irinotecan in children with refractory solid tumors. Cancer Chemother Pharmacol. 2006;57:15-24.
45. Kaufmann SH, Peereboom D, Buckwalter CA, et al. Cytotoxic effects of topotecan combined with various anticancer agents in human cancer cell lines. J Natl Cancer Inst. 1996; 88:734-741.
46. Janss AJ, Cnaan A, Zhao H, et al. Synergistic cytotoxicity of topoisomerase I inhibitors with alkylating agents and etoposide in human brain tumor cell lines. Anticancer Drugs. 1998; 9:641-652.
47. Bowers DC, Aquino VM, Leavey PJ, et al. Phase I study of oral cyclophosphamide and oral topotecan for children with recurrent or refractory solid tumors. Pediatr Blood Cancer. 2004;42:93-98.
48. Saylors RL, Stewart CF, Zamboni WC, et al. Phase I study of topotecan in combination with cyclophosphamide in pediatric patients with malignant solid tumors: a Pediatric Oncology Group Study. J Clin Oncol. 1998;16:945-952.
49. Saylors RL, 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;19:3463-3469.
50. Ashraf K, Shaikh F, Gibson P, et al. Treatment with topotecan plus cyclophosphamide in children with first relapse of neuroblastoma. Pediatr Blood Cancer. 2013. In press.
51. Park JR, Bagatell R, Hogarty M, et al. Children's Oncology Group's 2013 blueprint for research: Neuroblastoma. Pediatr Blood Cancer. 2013;60:985-993.
52. Thompson J, George EO, Poquette CA, et al. Synergy of topotecan in combination with vincristine for treatment of pediatric solid tumor xenografts. Clin Cancer Res. 1999;5: 3617-3631.
53. Kushner BH, Kramer K, Modak S, et al. Differential impact of high-dose cyclophosphamide, topotecan, and vincristine in clinical subsets of patients with chemoresistant neuroblastoma. Cancer. 2010;116:3054-3060.
54. Simon T, La ngler A, Berthold F, et al. Topotecan and etoposide in the treatment of relapsed high-risk neuroblastoma: results of a phase 2 trial. J Pediatr Hematol Oncol. 2007;29:101-106.
55. 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;98:2488-2494.
56. Di Giannatale A, Hugh KM, Dias N, et al. Phase II study of temozolomide in combination with topotecan (TOTEM) in relapsed or refractory neuroblastoma and other pediatric solid malignancies, A European ITCC study. J Clin Oncol. 2012;30, suppl-abstr 9517.
57. Houghton PJ, Stewart CF, Cheshire PJ, et al. Antitumor activity of temozolomide combined with irinotecan is partly independent of O6-methylguanine-DNA methyltransferase and mismatch repair phenotypes in xenograft models. Clin Cancer Res. 2000;6:4110-4118.
58. Middlemas DS, Stewart CF, Kirstein MN, et al. Biochemical correlates of temozolomide sensitivity in pediatric solid tumor xenograft models. Clin Cancer Res. 2000;6:998-1007.
59. Rubie H, Chisholm J, Defachelles AS, et al. Phase II study of temozolomide in relapsed or refractory high-risk neuroblastoma: a joint Socie te Francaise des Cancers de l'Enfant and United Kingdom Children Cancer Study Group-New Agents Group Study. J Clin Oncol. 2006;24:5259-5264.
60. Wagner LM, Crews KR, Stewart CF, et al. Reducing irinotecan-associated diarrhea in children. Pediatr Blood Cancer. 2008;50:201-207.
61. Innocenti F, Undevia SD, Iyer L, et al. Genetic variants in the UDP-glucuronosyltransferase 1A1 gene predict the risk of severe neutropenia of irinotecan. J Clin Oncol. 2004;22: 1382-1388.
62. Kushner BH, Kramer K, Modak S, et al. Irinotecan plus temozolomide for relapsed or refractory neuroblastoma. J Clin Oncol. 2006;24:5271-5276.
63. Park JR, Digiusto DL, Slovak M, et al. Adoptive transfer of chimeric antigen receptor re-directed cytolytic T lymphocyte clones in patients with neuroblastoma. Mol Ther. 2007;15: 825-833.
64. Wagner LM. Oral irinotecan for treatment of pediatric solid tumors: ready for prime time?. Pediatr Blood Cancer. 2010;54: 661-662.
65. Wagner LM, Villablanca JG, Stewart CF, et al. Phase I trial of oral irinotecan and temozolomide for children with relapsed high-risk neuroblastoma: a new approach to neuroblastoma therapy consortium study. J Clin Oncol. 2009; 27:1290-1296.
66. Cai W, Maldonado NV, Cui W, et al. Activity of irinotecan and temozolomide in the presence of O6-methylguanine-DNA methyltransferase inhibition in neuroblastoma preclinical models. Br J Cancer. 2010;103:1369-1379.
67. Wagner LM, McLendon RE, Yoon KJ, et al. Targeting methylguanine-DNA methyltransferase in the treatment of neuroblastoma. Clin Cancer Res. 2007;13:5418-5425.
68. Wagner LM, Danks MK. New therapeutic targets for the treatment of high-risk neuroblastoma. J Cell Biochem. 2009;107:46-57.
69. Kushner BH, Modak S, Kramer K, et al. Ifosfamide, carboplatin, and etoposide for neuroblastoma: a high-dose salvage regimen and review of the literature. Cancer. 2013; 119:665-667.
70. Hutchinson RJ, Sisson JC, Shapiro B, et al. 131-I-metaiodobenzylguanidine treatment in patients with refractory advanced neuroblastoma. Am J Clin Oncol. 1992;15:226-232.
71. Klingebiel T, Berthold F, Treuner J, et al. Metaiodobenzylguanidine (mIBG) in treatment of 47 patients with neuroblastoma: results of the German Neuroblastoma Trial. Med Pediatr Oncol. 1991;19:84-88.
72. Lashford LS, Lewis IJ, Fielding SL, et al. Phase I/II study of iodine 131 metaiodobenzylguanidine in chemoresistant neuroblastoma: a United Kingdom Children's Cancer Study Group investigation. J Clin Oncol. 1992;10:1889-1896.
73. Matthay KK, DeSantes K, Hasegawa B, et al. Phase I dose escalation of 131I-metaiodobenzylguanidine with autologous bone marrow support in refractory neuroblastoma. J Clin Oncol. 1998;16:229-236.
74. van Santen HM, de Kraker J, Vulsma T. Endocrine late effects from multi-modality treatment of neuroblastoma. Eur J Cancer. 2005;41:1767-1774.
75. Quach A, Ji L, Mishra V, et al. Thyroid and hepatic function after high-dose 131I-metaiodobenzylguanidine (131I-MIBG) therapy for neuroblastoma. Pediatr Blood Cancer. 2010;56: 191-201.
76. Gulenchyn KY, Yao X, Asa SL, et al. Radionuclide therapy in neuroendocrine tumours: a systematic review. Clin Oncol. 2012;24:294-308.
77. Weiss B, Vora A, Huberty J, et al. Secondary myelodysplastic syndrome and leukemia following 131I-metaiodobenzylguanidine therapy for relapsed neuroblastoma. J Pediatr Hematol Oncol. 2003;25:543-547.
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;25:1054-1060.
79. Polishchuk AL, Dubois SG, Haas-Kogan D, et al. Response, survival, and toxicity after iodine-131-metaiodobenzylguanidine therapy for neuroblastoma in preadolescents, adolescents, and adults. Cancer. 2011;117:4286-4293.
80. Kang TI, Brophy P, Hickeson M, et al. Targeted radiotherapy with submyeloablative doses of 131I-MIBG is effective for disease palliation in highly refractory neuroblastoma. J Pediatr Hematol Oncol. 2003;25:769-773.
81. de Kraker J, Hoefnagel KA, Verschuur AC, et al. Iodine-131- metaiodobenzylguanidine as initial induction therapy in stage 4 neuroblastoma patients over 1 year of age. Eur J Cancer. 2008;44:551-556.
82. Johnson K, McGlynn B, Saggio J, et al. Safety and efficacy of tandem 131I-metaiodobenzylguanidine infusions in relapsed/refractory neuroblastoma. Pediatr Blood Cancer. 2011;57: 1124-1129.
83. 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; 27:1020-1025.
84. Matthay KK, Tan JC, Villablanca JG, et al. Phase I dose escalation of iodine-131-metaiodobenzylguanidine with myeloablative chemotherapy and autologous stem-cell transplantation in refractory neuroblastoma: a new approaches to Neuroblastoma Therapy Consortium Study. J Clin Oncol. 2006;24:500-506.
85. Yanik GA, Levine JE, Matthay KK, et al. Pilot study of iodine-131-metaiodobenzylguanidine in combination with myeloablative chemotherapy and autologous stem-cell support for the treatment of neuroblastoma. J Clin Oncol. 2002;20:2142-2149.
86. Gaze MN, Chang Y-Ci, Flux GD, et al. Feasibility of dosimetry-based high-dose 131I-meta-iodobenzylguanidine with topotecan as a radiosensitizer in children with metastatic neuroblastoma. Cancer Biother Radiopharm. 2005;20: 195-199.
87. McCluskey AG, Boyd M, Pimlott SL, et al. Experimental treatment of neuroblastoma using [131I]meta-iodobenzylguanidine and topotecan in combination. Br J Radiol. 2008;81 (Spec No 1):S28-S35.
88. McCluskey AG, Boyd M, Ross SC, et al. [131I]metaiodobenzylguanidine and topotecan combination treatment of tumors expressing the noradrenaline transporter. Clin Cancer Res. 2005;11:7929-7937.
89. Mueller S, Yang X, Sottero TL, et al. Cooperation of the HDAC inhibitor vorinostat and radiation in metastatic neuroblastoma: efficacy and underlying mechanisms. Cancer Lett. 2011;306:223-229.
90. Boyd M, Ross S, Owens J, et al. Preclinical evaluation of no-carrier-added [131i]meta-iodobenzyl guanidine, for the treatment of tumours transfected with the noradrenaline transporter gene. Lett Drug Des Discov. 2004;1:50-57.
91. Matthay KK, Weiss B, Villablanca JG, et al. Dose escalation study of no-carrier-added 131I-metaiodobenzylguanidine for relapsed or refractory neuroblastoma: new approaches to neuroblastoma therapy consortium trial. J Nucl Med. 2012; 53:1155-1163.
92. Gains JE, Bomanji JB, Fersht NL, et al. 177Lu-DOTATATE molecular radiotherapy for childhood neuroblastoma. J Nucl Med. 2011;52:1041-1047.
93. Caussa L, Hijal T, Michon J, et al. Role of palliative radiotherapy in the management of metastatic pediatric neuroblastoma: a retrospective single-institution study. Int J Radiat Oncol Biol Phys. 2011;79:214-219.
94. Paulino AC. Palliative radiotherapy in children with neuroblastoma. Pediatr Hematol Oncol. 2003;20:111-117.
95. Modak S, Cheung N-KV. Disialoganglioside directed immunotherapy of neuroblastoma. Cancer Invest. 2007;25:67-77.
96. 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;363:1324-1334.
97. Cheung N-KV, Cheung IY, Kushner BH, et al. Murine anti-GD2 monoclonal antibody 3F8 combined with granulocytemacrophage colony-stimulating factor and 13-cis-retinoic acid in high-risk patients with stage 4 neuroblastoma in first remission. J Clin Oncol. 2012;30:3264-3270.
98. Simon T, Hero B, Faldum A, et al. Long term outcome of high-risk neuroblastoma patients after immunotherapy with antibody ch14.18 or oral metronomic chemotherapy. BMC Cancer. 2011;11:21.
99. Simon T, Hero B, Faldum A, et al. Consolidation treatment with chimeric anti-GD2-antibody ch14.18 in children older than 1 year with metastatic neuroblastoma. J Clin Oncol. 2004;22:3549-3557.
100. Delgado DC, Hank JA, Kolesar J, et al. Genotypes of NK cell KIR receptors, their ligands, and Fcg receptors in the response of neuroblastoma patients to Hu14.18-IL2 immunotherapy. Cancer Res. 2010;70:9554-9561.
101. Cheung NKV. FCGR2A polymorphism is correlated with clinical outcome after immunotherapy of neuroblastoma with anti-GD2 antibody and granulocyte macrophage colonystimulating factor. J Clin Oncol. 2006;24:2885-2890.
102. Navid F, Barfield RC, Handgretinger R, et al. A novel anti-GD2 monoclonal antibody (mAb), hu14.18K322A, in children with refractory or recurrent neuroblastoma: early-phase evaluation. J Clin Oncol. 2011;29, suppl-abstr 9523.
103. Uttenreuther-Fischer MM, Kru ger JA, Fischer P. Molecular characterization of the anti-idiotypic immune response of a relapse-free neuroblastoma patient following antibody therapy: a possible vaccine against tumors of neuroectodermal origin?. J Immunol. 2006;176:7775-7786.
104. Merchant MS, Baird K, Wexler LH, et al. Ipilimumab: first results of a phase I trial in pediatric patients with advanced solid tumors. J Clin Oncol. 2012;30, suppl abstr 9545.
105. Krishnadas DK, Shapiro T, Lucas K. Complete remission following decitabine/dendritic cell vaccine for relapsed neuroblastoma. Pediatrics. 2013;131:e336-e341.
106. Kanold J, Paillard C, Tchirkov A, et al. NK Cell immunotherapy for high-risk neuroblastoma relapse after haploidentical HSCT. Pediatr Blood Cancer. 2012;59:739-742.
107. Lee DW, Barrett DM, Mackall C, et al. The future is now: chimeric antigen receptors as new targeted therapies for childhood cancer. Clin Cancer Res. 2012;18:2780-2790.
108. 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;118:6050-6056.
109. Pule MA, Savoldo B, Myers GD, et al. Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma. Nat Med. 2008;14:1264-1270.
110. Jena B, Dotti G, Cooper LJN. Redirecting T-cell specificity by introducing a tumor-specific chimeric antigen receptor. Blood. 2010;116:1035-1044.
111. Kanold J, Paillard C, Tchirkov A, et al. Allogeneic or haploidentical HSCT for refractory or relapsed solid tumors in children: toward a neuroblastoma model. Bone Marrow Transplant. 2008;42(suppl 2):S25-S30.
112. Hale GA, Arora M, Ahn KW, et al. Allogeneic hematopoietic cell transplantation for neuroblastoma: the CIBMTR experience. Bone Marrow Transplant. 2013, Epub ahead of print. DOI: 10.1038/bmt.2012.284.
113. Lang P, Pfeiffer M, Mu ller I, et al. Haploidentical stem cell transplantation in patients with pediatric solid tumors: preliminary results of a pilot study and analysis of graft versus tumor effects. Klin Padiatr. 2006;218:321-326.
114. Jubert C, Wall DA, Grimley M, et al. Engraftment of unrelated cord blood after reduced-intensity conditioning regimen in children with refractory neuroblastoma: a feasibility trial. Bone Marrow Transplant. 2011;46:232-237.
115. Modak S, Cheung N-KV. Neuroblastoma: therapeutic strategies for a clinical enigma. Cancer Treat Rev. 2010; 36:307-317.
116. Tonini GP, Nakagawara A, Berthold F. Towards a turning point of neuroblastoma therapy. Cancer Lett. 2012;326: 128-134.
117. Garrido-Laguna I, Hidalgo M, Kurzrock R. The inverted pyramid of biomarker-driven trials. Nat Rev Clin Oncol. 2011;8:562-566.
118. Pugh TJ, Morozova O, Attiyeh EF, et al. The genetic landscape of high-risk neuroblastoma. Nat Genet. 2013; 45:279-284.
119. Otto T, Horn S, Brockmann M, et al. Stabilization of N-Myc is a critical function of Aurora A in human neuroblastoma. Cancer Cell. 2009;15:67-78.
120. Chesler L, Schlieve C, Goldenberg DD, et al. Inhibition of phosphatidylinositol 3-kinase destabilizes Mycn protein and blocks malignant progression in neuroblastoma. Cancer Res. 2006;66:8139-8146.
121. Puissant A, Frumm SM, Alexe G, et al. Targeting MYCN in neuroblastoma by BET bromodomain inhibition. Cancer Discov. 2013;3:308-323.
122. Janoueix-Lerosey I, Lequin D, Brugieres L, et al. Somatic and germline activating mutations of the ALK kinase receptor in neuroblastoma. Nature. 2008;455:967-970.
123. Mosse YP, Laudenslager M, Longo L, et al. Identification of ALK as a major familial neuroblastoma predisposition gene. Nature. 2008;455:930-935.
124. Chen Y, Takita J, Choi YL, et al. Oncogenic mutations of ALK kinase in neuroblastoma. Nature. 2008;455:971-974.
125. George RE, Sanda T, Hanna M, et al. Activating mutations in ALK provide a therapeutic target in neuroblastoma. Nature. 2008;455:975-978.
126. Mosse YP, Lim MS, Voss SD, et al. Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children's Oncology Group phase 1 consortium study. Lancet Oncology. 2013. [Epub ahead of print].
127. Bresler SC, Wood AC, Haglund EA, et al. Differential inhibitor sensitivity of anaplastic lymphoma kinase variants found in neuroblastoma. Sci Transl Med. 2011;3:108ra114.
128. Matthay KK, George RE, Yu AL. Promising therapeutic targets in neuroblastoma. Clin Cancer Res. 2012;18: 2740-2753.
129. Mosse YP, Lipsitz E, Fox E, et al. Pediatric phase I trial and pharmacokinetic study of MLN8237, an investigational oral selective small-molecule inhibitor of Aurora kinase A: a Children's Oncology Group Phase I Consortium study. Clin Cancer Res. 2012;18:6058-6064.
130. Hargrave DR, Pearson AD, Moreno L, et al. A phase I trial of AT9283 (a selective inhibitor of Aurora kinases) given for 72 hours every 21 days via intravenous infusion in children and adolescents with relapsed and refractory solid tumours. J Clin Oncoll. 2012;30, suppl-abstr 9542.
131. Bond M, Bernstein ML, Pappo A, et al. A phase II study of imatinib mesylate in children with refractory or relapsed solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2008;50:254-258.
132. Calafiore L, Amoroso L, Della Casa Alberighi O, et al. Twostage phase II study of imatinib mesylate in subjects with refractory or relapsing neuroblastoma. Ann Oncol. 2013; 24:1406-1413.
133. Minturn JE, Evans AE, Villablanca JG, et al. Phase I trial of lestaurtinib for children with refractory neuroblastoma: a new approaches to neuroblastoma therapy consortium study. Cancer Chemother Pharmacol. 2011;68:1057-1065.
134. Russell MR, Levin K, Rader J, et al. Combination therapy targeting the Chk1 and Wee1 kinases shows therapeutic efficacy in neuroblastoma. Cancer Res. 2013;73:776-784.
135. Sun W, Modak S. Emerging treatment options for the treatment of neuroblastoma: potential role of perifosine. Onco Targets Ther. 2012;5:21-29.
136. Segerstrom L, Fuchs D, Backman U, et al. The anti-VEGF antibody bevacizumab potently reduces the growth rate of high-risk neuroblastoma xenografts. Pediatr Res. 2006;60: 576-581.
137. Glade Bender JL, Adamson PC, Reid JM, et al. Phase I trial and pharmacokinetic study of bevacizumab in pediatric patients with refractory solid tumors: a Children's Oncology Group Study. J Clin Oncol. 2008;26:399-405.
138. Villablanca JG, London WB, Naranjo A, et al. Phase II study of oral capsular 4-hydroxyphenylretinamide (4-HPR/fenretinide) in pediatric patients with refractory or recurrent neuroblastoma: a report from the Children's Oncology Group. Clin Cancer Res. 2011;17:6858-6866.
139. Marachelian A, Kang MH, Hwang K, et al. Phase I study of fenretinide (4-HPR) oral powder in patients with recurrent or resistant neuroblastoma, New Approaches to Neuroblastoma Therapy (NANT) Consortium trial. J Clin Oncol. 2009;27, suppl-abstr 10009.
140. Kramer K, Kushner B, Heller G, et al. Neuroblastoma metastatic to the central nervous system. The Memorial Sloan-kettering Cancer Center experience and a literature review. Cancer. 2001;91:1510-1519.
141. Matthay KK, Brisse H, Couanet D, et al. Central nervous system metastases in neuroblastoma: radiologic, clinical, and biologic features in 23 patients. Cancer. 2003;98:155-165.
142. Carpenter EL, Mosse YP. Targeting ALK in neuroblastomapreclinical and clinical advancements. Nat Rev Clin Oncol. 2012;9:391-399.
143. Gustafson WC, Matthay KK. Progress towards personalized therapeutics: biologic-and risk-directed therapy for neuroblastoma. Expert Rev Neurother. 2011;11:1411-1423.
144. Couzin-Frankel J. Personalized medicine. Pushing the envelope in neuroblastoma therapy. Science. 2011;333:1569-1571.