Treatment of high-risk neuroblastoma

다국어 초록 (Multilingual 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 131I-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 treatmentrelated 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.

참고문헌 (Reference)

1 Kletzel M, "Treatment of high-risk neuroblastoma with triple-tandem high-dosetherapy and stem-cell rescue: results of the Chicago Pilot II Study" 20 : 2284-2292, 2002

2 Matthay KK, "Treatment of high-risk neuroblastoma with intensivechemotherapy, radiotherapy, autologous bone marrow transplantation,and 13-cis-retinoic acid" Children's Cancer Group 341 : 1165-1173, 1999

3 Sung KW, "Tandemhigh-dose chemotherapy and autologous stem cell rescue in patientsover 1 year of age with stage 4 neuroblastoma" 40 : 37-45, 2007

4 Goi K, "Successful tandem (autologous-cord blood) SCT in advanced neuroblastomaswith highly amplified MYCN" 46 : 835-839, 2011

5 Radich JP, "Second allogeneic transplantation after failure of first autologous transplantation" 6 : 272-279, 2000

6 Blaise D, "Reduced-intensity preparative regimen and allogeneic stem celltransplantation for advanced solid tumors" 103 : 435-441, 2004

7 Sung KW, "Reduced-intensity allogeneic stem cell transplantation for children with neuroblastoma who failed tandem autologous stem cell transplantation" 57 : 660-665, 2011

8 Yanik GA, "Pilot study of iodine-131-metaiodobenzylguanidine in combinationwith myeloablative chemotherapy and autologous stem-cell support forthe treatment of neuroblastoma" 20 : 2142-2149, 2002

9 Matthay KK, "Phase I dose escalation of iodine-131-metaiodobenzylguanidine withmyeloablative chemotherapy and autologous stem-cell transplantation inrefractory neuroblastoma: a new approaches to Neuroblastoma TherapyConsortium Study" 24 : 500-506, 2006

10 Matthay KK, "Phase I dose escalation of 131I-metaiodobenzylguanidine withautologous bone marrow support in refractory neuroblastoma" 16 : 229-236, 1998

11 Coldman AJ, "Neuroblastoma:influence of age at diagnosis, stage, tumor site, and sex on prognosis" 46 : 1896-1901, 1980

12 Cheung NK, "N7: a novel multi-modality therapy of high risk neuroblastoma(NB) in children diagnosed over 1 year of age" 36 : 227-230, 2001

13 Berthold F, "Myeloablative megatherapy with autologous stem-cell rescue versus oralmaintenance chemotherapy as consolidation treatment in patients withhigh-risk neuroblastoma: a randomised controlled trial" 6 : 649-658, 2005

14 Hobbie WL, "Late effects in survivors of tandem peripheral bloodstem cell transplant for high-risk neuroblastoma" 51 : 679-683, 2008

15 Frappaz D, "LMCE3 treatment strategy: results in 99 consecutively diagnosed stage 4neuroblastomas in children older than 1 year at diagnosis" 18 : 468-476, 2000

16 Pérez-Martínez A, "KIR-HLA receptor-ligand mismatch associated with a graftversus-tumor effect in haploidentical stem cell transplantation forpediatric metastatic solid tumors" 53 : 120-124, 2009

17 Takahashi H, "Iodine-131-metaiodobenzylguanidine therapy with reduced-intensity allogeneic stem cell transplantation in recurrent neuroblastoma" 50 : 676-678, 2008

18 Shimada H, "Histopathologic prognostic factors in neuroblastic tumors: definitionof subtypes of ganglioneuroblastoma and an age-linked classification ofneuroblastomas" 73 : 405-416, 1984

19 George RE, "High-risk neuroblastoma treated with tandemautologous peripheral-blood stem cell-supported transplantation: longtermsurvival update" 24 : 2891-2896, 2006

20 Toporski J, "High-dose iodine-131-metaiodobenzylguanidine with haploidenticalstem cell transplantation and posttransplant immunotherapy in childrenwith relapsed/refractory neuroblastoma" 15 : 1077-1085, 2009

21 Pritchard J, "High dose melphalan in the treatment of advanced neuroblastoma: resultsof a randomised trial (ENSG-1) by the European Neuroblastoma StudyGroup" 44 : 348-357, 2005

22 Lang P, "Haploidentical stem cell transplantation in patients with pediatric solidtumors: preliminary results of a pilot study and analysis of graft versustumor effects" 218 : 321-326, 2006

23 Appelbaum FR, "Haematopoietic cell transplantation as immunotherapy" 411 : 385-389, 2001

24 Marabelle A, "Graft-versus-tumour effect in refractory metastatic neuroblastoma" 39 : 809-810, 2007

25 Inoue M, "Graft-versus-tumor effect in a patient with advancedneuroblastomawho received HLA haplo-identical bone marrow transplantation" 32 : 103-106, 2003

26 Hirayama M, "Evidenceof graft-versus-tumor effect in refractory metastatic neuroblastoma" 82 : 142-144, 2006

27 Jubert C, "Engraftment of unrelated cord blood after reduced-intensity conditioning regimen in children with refractory neuroblastoma: a feasibility trial" 46 : 232-237, 2011

28 성기웅, "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)" 대한의학회 25 (25): 691-697, 2010

29 Joshi VV, "Correlation between morphologic and other prognostic markersof neuroblastoma. A study of histologic grade, DNA index, N-mycgene copy number, and lactic dehydrogenase in patients in the PediatricOncology Group" 71 : 3173-3181, 1993

30 Ladenstein R, "Comparison of auto versus allografting as consolidation of primarytreatments in advanced neuroblastoma over one year of age at diagnosis:report from the European Group for Bone Marrow Transplantation" 14 : 37-46, 1994

31 Look AT, "Clinical relevance of tumor cell ploidy and N-myc geneamplification in childhood neuroblastoma: a Pediatric Oncology Groupstudy" 9 : 581-591, 1991

32 Look AT, "CellularDNA content as a predictor of response to chemotherapy in infants withunresectable neuroblastoma" 311 : 231-235, 1984

33 de Lima M, "Bone marrow transplantation after failure of autologous transplant for non-Hodgkin's lymphoma" 19 : 121-127, 1997

34 Kyung-Ha Ryu, "Autologous Stem Cell Transplantation for the Treatment of Neuroblastoma in Korea" 대한의학회 18 (18): 242-247, 2003

35 Seeger RC, "Association of multiple copies of the N-myc oncogene with rapidprogression of neuroblastomas" 313 : 1111-1116, 1985

36 Yu AL, "Anti-GD2 antibody with GM-CSF, interleukin-2, andisotretinoin for neuroblastoma" 363 : 1324-1334, 2010

37 Brodeur GM, "Amplificationof N-myc in untreated human neuroblastomas correlates withadvanced disease stage" 224 : 1121-1124, 1984

38 Matthay KK, "Allogeneic versus autologous purged bone marrow transplantationfor neuroblastoma: a report from the Childrens Cancer Group" 12 : 2382-2389, 1994

39 Tsai T, "Allogeneic bone marrow transplantation in patients who relapse after autologous transplantation" 20 : 859-863, 1997

40 Philip T, "1070 myeloablative megatherapy procedures followed by stemcell rescue for neuroblastoma: 17 years of European experience andconclusions. European Group for Blood and Marrow Transplant RegistrySolid Tumour Working Party" 33 : 2130-2135, 1997