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http://dx.doi.org/10.3345/kjp.2012.55.4.115

Treatment of high-risk neuroblastoma  

Sung, Ki-Woong (Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine)
Publication Information
Clinical and Experimental Pediatrics / v.55, no.4, 2012 , pp. 115-120 More about this Journal
Abstract
Although high-dose chemotherapy and autologous stem cell transplantation (HDCT/autoSCT) have improved the prognosis for patients with high-risk neuroblastoma (NB), event-free survival rates remain in the range of 30 to 40%, which is unsatisfactory. To further improve outcomes, several clinical trials, including tandem HDCT/autoSCT, high-dose $^{131}I$-metaiodobenzylguanidine treatment, and immunotherapy with NB specific antibody, have been undertaken and pilot studies have reported encouraging results. Nonetheless, about half of high-risk NB patients still experience treatment failure and have no realistic chance for cure with conventional treatment options alone after relapse. Therefore, a new modality of treatment is warranted for these patients. In recent years, several groups of investigators have examined the feasibility and effectiveness of reduced-intensity allogeneic stem cell transplantation (RI alloSCT) for the treatment of relapsed/progressed NB. Although a graft-versus-tumor effect has not yet been convincingly demonstrated in the setting of relapsed NB, the strategy of employing RI alloSCT has provided hope that treatment-related mortality will be reduced and a therapeutic benefit will emerge. However, alloSCT for NB is still investigational and there remain many issues to be elucidated in many areas. At present, alloSCT is reserved for specific clinical trials testing the immunomodulatory effect against NB.
Keywords
Neuroblastoma; High-dose chemotherapy; Allogeneic stem cell transplantation;
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1 Sung KW, Lee SH, Yoo KH, Jung HL, Cho EJ, Koo HH, et al. Tandem high-dose chemotherapy and autologous stem cell rescue in patients over 1 year of age with stage 4 neuroblastoma. Bone Marrow Transplant 2007;40:37-45.   DOI   ScienceOn
2 Sung KW, Ahn HS, Cho B, Choi YM, Chung NG, Hwang TJ, et al. Efficacy of tandem high-dose chemotherapy and autologous stem cell rescue in patients over 1 year of age with stage 4 neuroblastoma: The Korean Society of Pediatric Hematology-Oncology Experience Over 6 Years (2000-2005). J Korean Med Sci 2010;25:691-7.   DOI   ScienceOn
3 Hobbie WL, Moshang T, Carlson CA, Goldmuntz E, Sacks N, Goldfarb SB, et al. Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma. Pediatr Blood Cancer 2008;51:679-83.   DOI   ScienceOn
4 Matthay KK, DeSantes K, Hasegawa B, Huberty J, Hattner RS, Ablin A, et al. Phase I dose escalation of 131I-metaiodobenzylguanidine with autologous bone marrow support in refractory neuroblastoma. J Clin Oncol 1998;16:229-36.   DOI
5 Matthay KK, Tan JC, Villablanca JG, Yanik GA, Veatch J, Franc B, 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;24:500-6.   DOI   ScienceOn
6 Yanik GA, Levine JE, Matthay KK, Sisson JC, Shulkin BL, Shapiro B, 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;20:2142-9.   DOI   ScienceOn
7 Cheung NK, Kushner BH, LaQuaglia M, Kramer K, Gollamudi S, Heller G, et al. N7: a novel multi-modality therapy of high risk neuroblastoma (NB) in children diagnosed over 1 year of age. Med Pediatr Oncol 2001;36:227-30.   DOI   ScienceOn
8 Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med 2010;363:1324-34.   DOI   ScienceOn
9 Ryu KH, Ahn HS, Koo HH, Kook H, Kim MK, Kim HK, et al. Autologous stem cell transplantation for the treatment of neuroblastoma in Korea. J Korean Med Sci 2003;18:242-7.   DOI
10 Philip T, Ladenstein R, Lasset C, Hartmann O, Zucker JM, Pinkerton R, et al. 1070 myeloablative megatherapy procedures followed by stem cell rescue for neuroblastoma: 17 years of European experience and conclusions. European Group for Blood and Marrow Transplant Registry Solid Tumour Working Party. Eur J Cancer 1997;33:2130-5.   DOI   ScienceOn
11 Appelbaum FR. Haematopoietic cell transplantation as immunotherapy. Nature 2001;411:385-9.   DOI   ScienceOn
12 Blaise D, Bay JO, Faucher C, Michallet M, Boiron JM, Choufi B, et al. Reduced-intensity preparative regimen and allogeneic stem cell transplantation for advanced solid tumors. Blood 2004;103:435-41.   DOI   ScienceOn
13 Inoue M, Nakano T, Yoneda A, Nishikawa M, Nakayama M, Yumura- Yagi K, et al. Graft-versus-tumor effect in a patient with advanced neuroblastoma who received HLA haplo-identical bone marrow transplantation. Bone Marrow Transplant 2003;32:103-6.   DOI   ScienceOn
14 Lang P, Pfeiffer M, Müller I, Schumm M, Ebinger M, Koscielniak E, 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;218:321-6.   DOI   ScienceOn
15 Marabelle A, Paillard C, Tchirkov A, Halle P, Chassagne J, Demeocq F, et al. Graft-versus-tumour effect in refractory metastatic neuroblastoma. Bone Marrow Transplant 2007;39:809-10.   DOI   ScienceOn
16 Look AT, Hayes FA, Shuster JJ, Douglass EC, Castleberry RP, Bowman LC, et al. Clinical relevance of tumor cell ploidy and N-myc gene amplification in childhood neuroblastoma: a Pediatric Oncology Group study. J Clin Oncol 1991;9:581-91.   DOI
17 Coldman AJ, Fryer CJ, Elwood JM, Sonley MJ. Neuroblastoma: influence of age at diagnosis, stage, tumor site, and sex on prognosis. Cancer 1980;46:1896-901.   DOI   ScienceOn
18 Brodeur GM, Seeger RC, Schwab M, Varmus HE, Bishop JM. Amplification of N-myc in untreated human neuroblastomas correlates with advanced disease stage. Science 1984;224:1121-4.   DOI
19 Seeger RC, Brodeur GM, Sather H, Dalton A, Siegel SE, Wong KY, et al. Association of multiple copies of the N-myc oncogene with rapid progression of neuroblastomas. N Engl J Med 1985;313:1111-6.   DOI   ScienceOn
20 Shimada H, Chatten J, Newton WA Jr, Sachs N, Hamoudi AB, Chiba T, et al. Histopathologic prognostic factors in neuroblastic tumors: definition of subtypes of ganglioneuroblastoma and an age-linked classification of neuroblastomas. J Natl Cancer Inst 1984;73:405-16.   DOI
21 Joshi VV, Cantor AB, Brodeur GM, Look AT, Shuster JJ, Altshuler G, 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.   DOI   ScienceOn
22 Tsai T, Goodman S, Saez R, Schiller G, Adkins D, Callander N, et al. Allogeneic bone marrow transplantation in patients who relapse after autologous transplantation. Bone Marrow Transplant 1997;20:859-63.   DOI   ScienceOn
23 Hirayama M, Azuma E, Araki M, Komada Y, Nakagawa A. Evidence of graft-versus-tumor effect in refractory metastatic neuroblastoma. Transplantation 2006;82:142-4.
24 Radich JP, Gooley T, Sanders JE, Anasetti C, Chauncey T, Appelbaum FR. Second allogeneic transplantation after failure of first autologous transplantation. Biol Blood Marrow Transplant 2000;6:272-9.   DOI   ScienceOn
25 de Lima M, van Besien KW, Giralt SA, Khouri IF, Mehra R, Andersson BS, et al. Bone marrow transplantation after failure of autologous transplant for non-Hodgkin's lymphoma. Bone Marrow Transplant 1997;19:121-7.   DOI   ScienceOn
26 Jubert C, Wall DA, Grimley M, Champagne MA, Duval M. Engraftment of unrelated cord blood after reduced-intensity conditioning regimen in children with refractory neuroblastoma: a feasibility trial. Bone Marrow Transplant 2011;46:232-7.   DOI   ScienceOn
27 Sung KW, Park JE, Chueh HW, Lee SH, Yoo KH, Koo HH, et al. Reduced- intensity allogeneic stem cell transplantation for children with neuroblastoma who failed tandem autologous stem cell transplantation. Pediatr Blood Cancer 2011;57:660-5.   DOI   ScienceOn
28 Takahashi H, Manabe A, Aoyama C, Kamiya T, Kato I, Takusagawa A, et al. Iodine-131-metaiodobenzylguanidine therapy with reducedintensity allogeneic stem cell transplantation in recurrent neuroblastoma. Pediatr Blood Cancer 2008;50:676-8.   DOI   ScienceOn
29 Frappaz D, Michon J, Coze C, Berger C, Plouvier E, Lasset C, et al. LMCE3 treatment strategy: results in 99 consecutively diagnosed stage 4 neuroblastomas in children older than 1 year at diagnosis. J Clin Oncol 2000;18:468-76.   DOI
30 Look AT, Hayes FA, Nitschke R, McWilliams NB, Green AA. Cellular DNA content as a predictor of response to chemotherapy in infants with unresectable neuroblastoma. N Engl J Med 1984;311:231-5.   DOI   ScienceOn
31 Matthay KK, Villablanca JG, Seeger RC, Stram DO, Harris RE, Ramsay NK, 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;341:1165-73.   DOI   ScienceOn
32 Berthold F, Boos J, Burdach S, Erttmann R, Henze G, Hermann J, 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.   DOI   ScienceOn
33 Pritchard J, Cotterill SJ, Germond SM, Imeson J, de Kraker J, Jones DR. 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.   DOI   ScienceOn
34 George RE, Li S, Medeiros-Nancarrow C, Neuberg D, Marcus K, Shamberger RC, et al. High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: longterm survival update. J Clin Oncol 2006;24:2891-6.   DOI   ScienceOn
35 Kletzel M, Katzenstein HM, Haut PR, Yu AL, Morgan E, Reynolds M, et al. Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue: results of the Chicago Pilot II Study. J Clin Oncol 2002;20:2284-92.   DOI   ScienceOn
36 Ladenstein R, Lasset C, Hartmann O, Klingebiel T, Bouffet E, Gadner H, et al. Comparison of auto versus allografting as consolidation of primary treatments in advanced neuroblastoma over one year of age at diagnosis: report from the European Group for Bone Marrow Transplantation. Bone Marrow Transplant 1994;14:37-46.
37 Toporski J, Garkavij M, Tennvall J, Ora I, Gleisner KS, Dykes JH, et al. High-dose iodine-131-metaiodobenzylguanidine with haploidentical stem cell transplantation and posttransplant immunotherapy in children with relapsed/refractory neuroblastoma. Biol Blood Marrow Transplant 2009;15:1077-85.   DOI   ScienceOn
38 Perez-Martinez A, Leung W, Munoz E, Iyengar R, Ramirez M, Vicario JL, et al. KIR-HLA receptor-ligand mismatch associated with a graftversus- tumor effect in haploidentical stem cell transplantation for pediatric metastatic solid tumors. Pediatr Blood Cancer 2009;53:120-4.   DOI   ScienceOn
39 Matthay KK, Seeger RC, Reynolds CP, Stram DO, O'Leary MC, Harris RE, et al. Allogeneic versus autologous purged bone marrow transplantation for neuroblastoma: a report from the Childrens Cancer Group. J Clin Oncol 1994;12:2382-9.   DOI
40 Goi K, Inukai T, Honna H, Akahane K, Hirose K, Kuroda I, et al. Successful tandem (autologous-cord blood) SCT in advanced neuroblastomas with highly amplified MYCN. Bone Marrow Transplant 2011;46:835-9.   DOI   ScienceOn