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Current Cancer Therapy Reviews

Editor-in-Chief

ISSN (Print): 1573-3947
ISSN (Online): 1875-6301

Review Article

High-Risk Neuroblastoma: Poor Outcomes Despite Aggressive Multimodal Therapy

Author(s): Adil Abdelhamed Abbas* and Alaa Mohammed Noor Samkari

Volume 18, Issue 1, 2022

Published on: 05 August, 2021

Page: [14 - 40] Pages: 27

DOI: 10.2174/1573394717666210805114226

Price: $65

Abstract

Neuroblastoma (NBL) is a highly malignant embryonal tumor that originates from the primordial neural crest cells. NBL is the most common tumor in infants and the most common extracranial solid tumor in children. The tumor is more commonly diagnosed in children of 1-4 years of age. NBL is characterized by enigmatic clinical behavior that ranges from spontaneous regression to an aggressive clinical course leading to frequent relapses and death. Based on the likelihood of progression and relapse, the International Neuroblastoma Risk Group classification system categorized NBL into very low risk, low risk, intermediate risk, and high risk (HR) groups. HR NBL is defined based on the patient's age (> 18 months), disease metastasis, tumor histology, and MYCN gene amplification. HR NBL is diagnosed in nearly 40% of patients, mainly those > 18 months of age, and is associated with aggressive clinical behavior. Treatment strategies involve the use of intensive chemotherapy (CTR), surgical resection, high dose CTR with hematopoietic stem cell support, radiotherapy, biotherapy, and immunotherapy with Anti-ganglioside 2 monoclonal antibodies. Although HR NBL is now better characterized and aggressive multimodal therapy is applied, the outcomes of treatment are still poor, with overall survival and event-free survival of approximately 40% and 30% at 3-years, respectively. The short and long-term side effects of therapy are tremendous. HR NBL carries a high mortality rate accounting for nearly 15% of pediatric cancer deaths. However, most mortalities are attributed to the high frequency of disease relapse (50%) and disease reactiveness to therapy (20%). Newer treatment strategies are therefore urgently needed. Recent discoveries in the field of biology and molecular genetics of NBL have led to the identification of several targets that can improve the treatment results. In this review, we discuss the different aspects of the epidemiology, biology, clinical presentations, diagnosis, and treatment of HR NBL, in addition to the recent developments in the management of the disease.

Keywords: Chemotherapy, dinutuximab, isotretinoin, neuroblastoma, MYCN gene, risk stratification, stem cell transplantation, targeted therapy.

Graphical Abstract
[1]
Van Noesel MM, Versteeg R. Pediatric neuroblastomas: Genetic and epigenetic ‘danse macabre’. Gene 2004; 325: 1-15.
[2]
Papaioannou G, McHugh K. Neuroblastoma in childhood: review and radiological findings. Cancer Imaging 2005; 5(1): 116-27.
[http://dx.doi.org/10.1102/1470-7330.2005.0104] [PMID: 16305949]
[3]
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; 21(3): 181-9.
[http://dx.doi.org/10.1097/00043426-199905000-00005] [PMID: 10363850]
[4]
Dubois SG, London WB, Zhang Y, et al. Lung metastases in neuroblastoma at initial diagnosis: A report from the International Neuroblastoma Risk Group (INRG) project. Pediatr Blood Cancer 2008; 51(5): 589-92.
[http://dx.doi.org/10.1002/pbc.21684] [PMID: 18649370]
[5]
Kushner BH, Gilbert F, Helson L. Familial neuroblastoma: Case reports, literature review, and etiologic considerations. Cancer 1986; 57: 1887.
[6]
Hallett A, Traunecker H. A review and update on neuroblastoma J Pediatr Child Health. 2012; 22(22): 103.
[7]
Maris JM. Recent advances in neuroblastoma. N Engl J Med 2010; 362(23): 2202-11.
[http://dx.doi.org/10.1056/NEJMra0804577] [PMID: 20558371]
[8]
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(2): 289-97.
[http://dx.doi.org/10.1200/JCO.2008.16.6785] [PMID: 19047291]
[9]
Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol 2015; 33(27): 3008-17.
[http://dx.doi.org/10.1200/JCO.2014.59.4648] [PMID: 26304901]
[10]
Bowen KA, Chung DH. Recent advances in neuroblastoma. Curr Opin Pediatr 2009; 21(3): 350-6.
[http://dx.doi.org/10.1097/MOP.0b013e32832b1240] [PMID: 19381091]
[11]
Aravindan N, Jain D, Somasundaram DB, Herman TS, Aravindan S. Cancer stem cells in neuroblastoma therapy resistance. Cancer Drug Resist 2019; 2: 948-67.
[http://dx.doi.org/10.20517/cdr.2019.72] [PMID: 31867574]
[12]
Ganeshan VR, Schor NF. Pharmacologic management of high-risk neuroblastoma in children. Paediatr Drugs 2011; 13(4): 245-55.
[http://dx.doi.org/10.2165/11591630-000000000-00000] [PMID: 21692548]
[13]
Berthold F, Spix C, Kaatsch P, Lampert F. Incidence, survival, and treatment of localized and metastatic neuroblastoma in germany 1979-2015. Paediatr Drugs 2017; 19(6): 577-93.
[http://dx.doi.org/10.1007/s40272-017-0251-3] [PMID: 28786082]
[14]
Richards RM, Sotillo E, Majzner RG. CAR T cell therapy for neuroblastoma. Frontiers in Immunology 2018; 9: 2380.
[15]
Rha SE, Byun JY, Jung SE, Chun HJ, Lee HG, Lee JM. Neurogenic tumors in the abdomen: Tumor types and imaging characteristics. Radiographics 2003; 23(1): 29-43.
[http://dx.doi.org/10.1148/rg.231025050] [PMID: 12533638]
[16]
Chow EJ, Friedman DL, Mueller BA. Maternal and perinatal characteristics in relation to neuroblastoma. Cancer 2007; 109(5): 983-92.
[http://dx.doi.org/10.1002/cncr.22486] [PMID: 17285600]
[17]
Imbach P. Neuroblastoma.Pediatric Oncology, A comprehensive Guide New York: Springer –Verlag Berlin Heidelberg Publications. 2004; pp. 119-28.
[18]
Ross JA, Davies SM. Screening for neuroblastoma: Progress and pitfalls. Cancer Epidemiol Biomarkers Prev 1999; 8(2): 189-94.
[PMID: 10067819]
[19]
Kesrouani A, Duchatel F, Seilanian M, Muray JM. Prenatal Diagnosis of Adrenal Neuroblastoma by Ultrasound: A Report of Two Cases and Review of the Literature 1999.
[20]
Stiller CA, Parkin DM. International variations in the incidence of neuroblastoma. Int J Cancer 1992; 52(4): 538-43.
[http://dx.doi.org/10.1002/ijc.2910520407] [PMID: 1399133]
[21]
Vénat-Bouvet L, Le Brun-Ly V, Martin J, Gasnier O, Falkowsky S, Tubiana-Mathieu N. Long-Term Survival in Adult Neuroblastoma with Multiple Recurrences. Case Rep Oncol 2010; 3(1): 45-8.
[http://dx.doi.org/10.1159/000286142] [PMID: 20740158]
[22]
Lonergan GJ, Schwab CM, Suarez ES, Carlson CL. Neuroblastoma, ganglioneuroblastoma, and ganglioneuroma: Radiologic- pathologic correlation. Radiographics 2002; 22(4): 911-34.
[http://dx.doi.org/10.1148/radiographics.22.4.g02jl15911] [PMID: 12110723]
[23]
Schilling FH, Bihl H, Jacobsson H, Ambros PF, Martinsson T, Borgström P, et al. al. Combined (111) In-pentetreotide scintigraphy and (123) I-mIBG scintigraphy in neuroblastoma provides prognostic information. Med Pediatr Oncol 2000; 35: 688-91.
[24]
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; 20(1): 27-32.
[http://dx.doi.org/10.1007/s00383-003-1070-x] [PMID: 14689211]
[25]
Tolbert VP, Matthay KK. Neuroblastoma: Clinical and biological approach to risk stratification and treatment. Cell Tissue Res 2018; 372(2): 195-209.
[http://dx.doi.org/10.1007/s00441-018-2821-2] [PMID: 29572647]
[26]
Vo KT, Matthay KK, Neuhaus J, et al. Clinical, biologic, and prognostic differences on the basis of primary tumor site in neuroblastoma: A report from the international neuroblastoma risk group project. J Clin Oncol 2014; 32(28): 3169-76.
[http://dx.doi.org/10.1200/JCO.2014.56.1621] [PMID: 25154816]
[27]
Morgenstern DA, London WB, Stephens D, Volchenboum SL, Simon T, Nakagawara A, et al. Prognostic significance of pattern and burden of metastatic disease in patients with stage 4 neuroblastoma: A study from the International Neuroblastoma Risk Group database. Eur J Cancer 2016.
[28]
Colon NC, Chung DH. Neuroblastoma. Adv Pediatr 2011; 58: 279-311.
[29]
Morgenstern DA, Pötschger U, Moreno L, et al. Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study. Pediatr Blood Cancer 2018; 65(11): e27363.
[http://dx.doi.org/10.1002/pbc.27363] [PMID: 30015396]
[30]
Hann HW, 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(6): 2843-8.
[PMID: 3986811]
[31]
Trigg RM, Shaw JA, Turner SD. Opportunities and challenges of circulating biomarkers in neuroblastoma. Open Biol 2019; 9(5): 190056.
[http://dx.doi.org/10.1098/rsob.190056] [PMID: 31088252]
[32]
Perakis S, Speicher MR. Emerging concepts in liquid biopsies. 2017; 15: 75.
[33]
Montani F, Bianchi F. Circulating Cancer Biomarkers: The Macro-revolution of the Micro-RNA. EBioMedicine 2016; 5: 4-6.
[http://dx.doi.org/10.1016/j.ebiom.2016.02.038] [PMID: 27077096]
[34]
Verly IR, van Kuilenburg AB, Abeling NG, Goorden SM, Fiocco M, Vaz FM, et al. Catecholamine profiles at diagnosis: Increased diagnostic sensitivity and correlation with biological and clinical features with biological and clinical features in neuroblastoma patients. Eur J Cancer 2017; 72: 235-43.
[http://dx.doi.org/10.1016/j.ejca.2016.12.002] [PMID: 28061374]
[35]
Tsubono Y, Hisamichi S. A halt to neuroblastoma screening in Japan. N Engl J Med 2004; 350(19): 2010-1.
[http://dx.doi.org/10.1056/NEJM200405063501922] [PMID: 15128908]
[36]
Meyer JS, Harty MP, Khademian Z. Imaging of neuroblastoma and Wilms’ tumor. Magn Reson Imaging Clin N Am 2002; 10(2): 275-302.
[http://dx.doi.org/10.1016/S1064-9689(01)00010-1] [PMID: 12424947]
[37]
Kushner BH. Neuroblastoma: a disease requiring a multitude of imaging studies. J Nucl Med 2004; 45(7): 1172-88. [PMID: 15235064].
[PMID: 15235064]
[38]
Hiorns MP, Owens CM. Radiology of neuroblastoma in children. Eur Radiol 2001; 11(10): 2071-81.
[http://dx.doi.org/10.1007/s003300100931] [PMID: 11702143]
[39]
Wu YH, Song B, Xu J, Chen WX, Zhao WX, Jia R, et al. Retroperitoneal neoplasms within the perirenal space in infants and children: differentiation of renal and non-renal origin in enhanced CT images. Eur J Radiol 2010; 75(3)
[40]
Shulkin BL, Hutchinson RJ, Castle VP, Yanik GA, Shapiro B, Sisson JC. Neuroblastoma: positron emission tomography with 2-[fluorine-18]-fluoro-2-deoxy-D-glucose compared with metaiodobenzylguanidine scintigraphy. Radiology 1996; 199(3): 743-50.
[http://dx.doi.org/10.1148/radiology.199.3.8637999] [PMID: 8637999]
[41]
Sharp SE, Shulkin BL, Gelfand MJ, Salisbury S, Furman WL. MIBG scintigraphy and 18F-FDG PET in neuroblastoma. J Nucl Med 2009; 50: 1237.
[42]
Albers AR, O’Dorisio MS, Balster DA, Caprara M, Gosh P, Chen F, et al. Somatostatin receptor gene expression in neuroblastoma. Regul Pept 2000; 88: 61-73.
[43]
Brodeur GM, Seeger RC, Schwab M, Vamus HE, Bishop JM. Amplification of NMYC gene in untreated human Neuroblastoma correlated with advanced disease stage. Science 224: 1121-4.
[44]
Molenaar JJ, Koster J, Zwijnenburg DA, Sluis PV, Valentijn LJ, van der Ploeg I, et al. Sequencing of neuroblastoma identifies chromothripsis and defects in neuritogenesis genes. Nature 2012; 483-593.
[45]
Devoto M, Specchia C, Laudenslager M, et al. Genome-wide linkage analysis to identify genetic modifiers of ALK mutation penetrance in familial neuroblastoma. Hum Hered 2011; 71(2): 135-9.
[http://dx.doi.org/10.1159/000324843] [PMID: 21734404]
[46]
Bagatell R, Cohn SL. Genetic discoveries and treatment advances in neuroblastoma. Curr Opin Pediatr 2016; 28(1): 19-25.
[http://dx.doi.org/10.1097/MOP.0000000000000296] [PMID: 26576010]
[47]
Shimada H, Ambros IM, Dehner LP, Hata J, Joshi VV, Roald B. Terminology and morphologic criteria of neuroblastic tumors: recommendations by the International Neuroblastoma Pathology Committee. Cancer 1999; 86(2): 349-63.
[http://dx.doi.org/10.1002/(SICI)1097-0142(19990715)86:2<349::AID-CNCR20>3.0.CO;2-Y] [PMID: 10421272]
[48]
Umehara S, Nakagawa A, Matthay KK, et al. Histopathology defines prognostic subsets of ganglioneuroblastoma, nodular. Cancer 2000; 89(5): 1150-61.
[http://dx.doi.org/10.1002/1097-0142(20000901)89:5<1150::AID-CNCR25>3.0.CO;2-7] [PMID: 10964346]
[49]
Katenkamp D, Stiller D, Holzhausen HJ. Morphology of neuroblastoma. Light and electron microscopic studies as a contribution on diagnosis and differential diagnosis. Zentralbl Allg Pathol 1983; 127(3-4): 207-18.
[PMID: 6349176]
[50]
Duijkers FAM, Gaal J, Meijerink JP, et al. High anaplastic lymphoma kinase immunohistochemical staining in neuroblastoma and ganglioneuroblastoma is an independent predictor of poor outcome. Am J Pathol 2012; 180(3): 1223-31.
[51]
Bielle F, Fréneaux P, Jeanne-Pasquier C, Maran-Gonzalez A, Rousseau A, Lamant L, et al. PHOX2B immunolabeling: A novel tool for the diagnosis of undifferentiated neuroblastomas among childhood small round blue-cell tumors. Am J Surg Pathol 2012; 36: 1141-9.
[52]
Cheung IY, Barber D, Cheung NK. Detection of microscopic neuroblastoma in marrow by histology, immunocytology, and reverse transcription-PCR of multiple molecular markers. Clin Cancer Res 1998; 4(11): 2801-5. [PMID: 9829745].
[PMID: 9829745]
[53]
Burchill SA, Beiske K, Shimada H, et al. Recommendations for the standardization of bone marrow disease assessment and reporting in children with neuroblastoma on behalf of the International Neuroblastoma Response Criteria Bone Marrow Working Group. Cancer 2017; 123(7): 1095-105.
[http://dx.doi.org/10.1002/cncr.30380] [PMID: 27984660]
[54]
Rastogi P, Naseem S, Varma N, et al. Bone marrow involvement in neuroblastoma: A study of hemato-morphological features. Indian J Hematol Blood Transfus 2015; 31(1): 57-60.
[http://dx.doi.org/10.1007/s12288-014-0405-1] [PMID: 25548446]
[55]
Okamatsu C, London WB, Naranjo A, et al. Clinicopathological characteristics of ganglioneuroma and ganglioneuroblastoma: a report from the CCG and COG. Pediatr Blood Cancer 2009; 53(4): 563-9.
[http://dx.doi.org/10.1002/pbc.22106] [PMID: 19530234]
[56]
Shimada H, Chatten J, Newton WA Jr, 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(2): 405-16.
[http://dx.doi.org/10.1093/jnci/73.2.405] [PMID: 6589432]
[57]
Lu D, Liu J, Chen Y, Chen F, Yang H. Primary Cervical Ganglioneuroblastoma: A Case Report. Medicine (Baltimore 2018; 97(12)
[58]
Robertson HE. The Ganglioblastoma, a special type in the System of Neuromas. Virchows Arch 1915; 63: 147-68.
[http://dx.doi.org/10.1007/BF01949097]
[59]
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(8): 1466-77.
[http://dx.doi.org/10.1200/JCO.1993.11.8.1466] [PMID: 8336186]
[60]
Monclair T, Brodeur GM, Ambros PF, et al. The International Neuroblastoma Risk Group (INRG) staging system: an INRG Task Force report. J Clin Oncol 2009; 27(2): 298-303.
[http://dx.doi.org/10.1200/JCO.2008.16.6876] [PMID: 19047290]
[61]
SEER1 Cancer Statistics Review. National Cancer Institute, Surveillance, Epidemiology and End result program 1999.
[62]
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(9): 1471-82.
[http://dx.doi.org/10.1038/sj.bjc.6605014] [PMID: 19401703]
[63]
Beiske K, Burchill SA, Cheung IY, et al. Consensus criteria for sensitive detection of minimal neuroblastoma cells in bone marrow, blood and stem cell preparations by immunocytology and QRT-PCR: recommendations by the International Neuroblastoma Risk Group Task Force. Br J Cancer 2009; 100(10): 1627-37.
[http://dx.doi.org/10.1038/sj.bjc.6605029] [PMID: 19401690]
[64]
Irwin MS, Park JR. Neuroblastoma: paradigm for precision medicine. Pediatr Clin North Am 2015; 62(1): 225-56.
[http://dx.doi.org/10.1016/j.pcl.2014.09.015] [PMID: 25435121]
[65]
Cheung NK, Dyer MA. Neuroblastoma: developmental biology, cancer genomics and immunotherapy. Nat Rev Cancer 2013; 13(6): 397-411.
[http://dx.doi.org/10.1038/nrc3526] [PMID: 23702928]
[66]
Pearson AD, Pinkerton CR, Lewis IJ, Imeson J, Ellershaw C, Machin D. High-dose rapid and standard induction chemotherapy for patients aged over 1 year with stage 4 neuroblastoma: A randomised trial. Lancet Oncol 2008; 9(3): 247-56.
[http://dx.doi.org/10.1016/S1470-2045(08)70069-X] [PMID: 18308250]
[67]
Rujkijyanont P, Photia A, Traivaree C, et al. Clinical outcomes and prognostic factors to predict treatment response in high risk neuroblastoma patients receiving topotecan and cyclophosphamide containing induction regimen: A prospective multicenter study. BMC Cancer 2019; 19(1): 961.
[http://dx.doi.org/10.1186/s12885-019-6186-z] [PMID: 31619207]
[68]
Baker DL, Schmidt ML, Cohn SL, et al. Outcome after reduced chemotherapy for intermediate-risk neuroblastoma. N Engl J Med 2010; 363(14): 1313-23.
[http://dx.doi.org/10.1056/NEJMoa1001527] [PMID: 20879880]
[69]
Murphy JM, La Quaglia MP. Advances in the surgical treatment of neuroblastoma: a review. Eur J Pediatr Surg 2014; 24(6): 450-6.
[http://dx.doi.org/10.1055/s-0034-1396421] [PMID: 25486413]
[70]
Zwaveling S, Tytgat GA, van der Zee DC, Wijnen MH, Heij HA. Is complete surgical resection of stage 4 neuroblastoma a prerequisite for optimal survival or may >95 % tumour resection suffice? Pediatr Surg Int 2012; 28(10): 953-9.
[http://dx.doi.org/10.1007/s00383-012-3109-3] [PMID: 22722825]
[71]
Von Schweinitz D, Hero B, Berthold F. The impact of surgical radicality on outcome in childhood neuroblastoma. Eur J Pediatr Surg 2002; 6: 402-9.
[72]
Luo YB, Cui XC, Yang L, Zhang D, Wang JX. Advances in the Surgical Treatment of Neuroblastoma. Chin Med J 2018.
[73]
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(4): 348-57.
[http://dx.doi.org/10.1002/pbc.20219] [PMID: 15546135]
[74]
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(9): 649-58.
[http://dx.doi.org/10.1016/S1470-2045(05)70291-6] [PMID: 16129365]
[75]
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. N Engl J Med 1999; 341(16): 1165-73.
[http://dx.doi.org/10.1056/NEJM199910143411601] [PMID: 10519894]
[76]
Matthay KK, Reynolds CP, Seeger RC, Shimada H, Adkins ES, Haas-Kogan D, et al. 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(7): 1007-3.
[77]
Ladenstein R, Pötschger U, Hartman O, Pearson ADJ, Klingebiel T, Castel V, et al. years of high-dose therapy and SCT for neuroblastoma in Europe: Lessons from more than 4000 procedures. Bone Marrow Transplant 2008.
[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(9): 1054-60.
[http://dx.doi.org/10.1200/JCO.2006.09.3484] [PMID: 17369569]
[79]
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; 14(10): 999-1008.
[http://dx.doi.org/10.1016/S1470-2045(13)70309-7] [PMID: 23890779]
[80]
Ladenstein R, Pötschger U, Pearson ADJ, Brock P, Luksch R, Castel V, et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): An international, randomized, multi-arm, open-label, phase 3 trial. Lancet Oncol 2017; 18(4): 500-14.
[81]
Park JR, Kreissman SG, London WB, et al. Effect of tandem autologous stem cell transplant vs single transplant on event-free survival in patients with high-risk neuroblastoma: A Randomized clinical trial. JAMA 2019; 322(8): 746-55.
[http://dx.doi.org/10.1001/jama.2019.11642] [PMID: 31454045]
[82]
Simon T, Bongartz R, Hero B, Schmidt M, Müller RP, Berthold F. Intensified external beam radiation therapy improves the outcome of stage 4 neuroblastoma in children >1 year with residual local disease. Strahlenther Onkol 2006; 182(7): 389-94.
[83]
Wagner LM, Danks MK. New therapeutic targets for the treatment of high-risk neuroblastoma. J Cell Biochem 2009; 107(1): 46-57.
[http://dx.doi.org/10.1002/jcb.22094] [PMID: 19277986]
[84]
Modak S, Cheung NK. Neuroblastoma: Therapeutic strategies for a clinical enigma. Cancer Treat Rev Jun 36(4): 307-17.
[85]
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(14): 1324-34.
[http://dx.doi.org/10.1056/NEJMoa0911123] [PMID: 20879881]
[86]
Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, et al. New response evaluation criteria in solid tumours. European J Cancer 2009; 45(2): 228-47.
[87]
Wahl RL, Jacene H, Kasamon Y, Lodge MA. From RECIST to PERCIST: Evolving considerations for PET response criteria in solid tumors. J Nucl Med 2009; 50(1): 122S-50S.
[http://dx.doi.org/10.2967/jnumed.108.057307] [PMID: 19403881]
[88]
Wang X, Wang L, Su Y, Yue Z, T, Zhao W, Zhao Q, et al. Plasma cell free DNA quantification is highly correlated with tumor burden in children with neuroblastoma. Cancer Med 2018; 7: 3022-30.
[89]
Cole KA, Maris JM. New Strategies in Refractory and Recurrent Neuroblastoma: Translational Opportunities to Impact Patient Outcome. Clin Cancer Res 2012; 18: 2423-8.
[90]
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; 35(5): 337-47.
[http://dx.doi.org/10.1097/MPH.0b013e318299d637] [PMID: 23703550]
[91]
Moreno L, Rubie H, Varo A, et al. Outcome of children with relapsed or refractory neuroblastoma: A meta-analysis of ITCC/SIOPEN European phase II clinical trials. Pediatr Blood Cancer 2017; 64(1): 25-31.
[http://dx.doi.org/10.1002/pbc.26192] [PMID: 27555472]
[92]
London WB, Bagatell R, Weigel BJ, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children’s Oncology Group early-phase trials. Cancer 2017; 123: 4914-23.
[93]
Garaventa A, Gambini C, Villavecchia G, et al. Second malignancies in children with neuroblastoma after combined treatment with 131I-metaiodobenzylguanidine. Cancer 2003; 97(5): 1332-8.
[http://dx.doi.org/10.1002/cncr.11167] [PMID: 12599242]
[94]
Manole BA, Wakefield DV, Doubrovin M, Shulkin BB, Merchant TE, Davidoff AM, et al. Management of local-regional failure in children with high-risk neuroblastoma. A single institution experience. Int J Radiat Oncol Biol Phys 2017; 99 E571.doi.org/ 10.1016/
[95]
Eleveld TF, Oldridge DA, Bernard V, et al. Relapsed neuroblastomas show frequent RAS-MAPK pathway mutations. Nat Genet 2015; 47(8): 864-71.
[http://dx.doi.org/10.1038/ng.3333] [PMID: 26121087]
[96]
Padovan-Merhar OM, Raman P, Ostrovnaya I, et al. Enrichment of targetable mutations in the relapsed neuroblastoma genome. PLoS Genet 2016; 12(12): e1006501.
[http://dx.doi.org/10.1371/journal.pgen.1006501] [PMID: 27997549]
[97]
Kushner BH, Ostrovnaya I, Cheung IY, et al. Prolonged progression-free survival after consolidating second or later remissions of neuroblastoma with anti-GD2 immunotherapy and isotretinoin: A prospective phase II study. Oncoimmunology 2015; 4
[98]
London WB2, Frantz CN, Campbell LA, Seeger RC, Brumback BA, Cohn SL. 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: 3808-15.
[99]
Ashraf K, Shaikh F, Gibson P, Baruchel S, Irwin MS. Treatment with topotecan plus cyclophosphamide in children with first relapse of neuroblastoma. Pediatr Blood Cancer 2013; 60(10): 1636-41.
[http://dx.doi.org/10.1002/pbc.24587] [PMID: 23650219]
[100]
Kushner BH, Kramer K, Modak S, Qin LX, Cheung NKV. Differential impact of high-dose cyclophosphamide, topotecan, and vincristine in clinical subsets of patients with chemoresistant neuroblastoma. Cancer 2010; 116: 3045-60.
[101]
Kushner BH, Kramer K, Modak S, Cheung NKV. Irinotecan plus temozolomide for relapsed or refractory neuroblastoma. J Clin Oncol 2006; 24(33): 5271-6.
[http://dx.doi.org/10.1200/JCO.2006.06.7272] [PMID: 17114661]
[102]
Modak S, Kushner BH, Basu E, Roberts SS, Cheung NKV. Combination of bevacizumab, irinotecan, and temozolomide for refractory or relapsed neuroblastoma: Results of a phase II study. Pediatr Blood Cancer 2017; 64
[103]
Amoroso L, Erminio G, Makin G, et al. Topotecan-vincristine- doxorubicin in stage 4 high-risk neuroblastoma patients failing to achieve a complete metastatic response to rapid cojec: A siopen study. Cancer Res Treat 2018; 50(1): 148-55.
[http://dx.doi.org/10.4143/crt.2016.511] [PMID: 28324923]
[104]
Kushner BH, Modak S, Kramer K, Basu EM, Roberts SS, Cheung NK. Ifosfamide, carboplatin, and etoposide for neuroblastoma: A high-dose salvage regimen and review of the literature. Cancer 2013; 119(3): 665-71.
[http://dx.doi.org/10.1002/cncr.27783] [PMID: 22951749]
[105]
Rufini V, Calcagni ML, Baum RP. Imaging of neuroendocrine tumors. Semin Nucl Med 2006; 36(3): 228-47.
[http://dx.doi.org/10.1053/j.semnuclmed.2006.03.007] [PMID: 16762613]
[106]
Eisenhofer G, Pacak K, Goldstein DS, Chen C, Shulkin B. 123I-MIBG scintigraphy of catecholamine systems: Impediments to applications in clinical medicine. Eur J Nucl Med 2000; 27(5): 611-2.
[http://dx.doi.org/10.1007/s002590000263] [PMID: 10853820]
[107]
Fitzgerald PA, Goldsby RE, Huberty JP, et al. Malignant pheochromocytomas and paragangliomas: A phase II study of therapy with high-dose 131I-metaiodobenzylguanidine (131I-MIBG). Ann N Y Acad Sci 2006; 1073: 465-90.
[http://dx.doi.org/10.1196/annals.1353.050] [PMID: 17102115]
[108]
Treuner J, Klingebiel T, Feine U, et al. Clinical experiences in the treatment of neuroblastoma with 131I-metaiodobenzylguanidine. Pediatr Hematol Oncol 1986; 3(3): 205-16.
[http://dx.doi.org/10.3109/08880018609031220] [PMID: 2484676]
[109]
Guller U, Turek J, Eubanks S, Delong ER, Oertli D, Feldman JM. Detecting pheochromocytoma: defining the most sensitive test. Ann Surg 2006; 243(1): 102-7.
[http://dx.doi.org/10.1097/01.sla.0000193833.51108.24] [PMID: 16371743]
[110]
Giammarile F, Chiti A, Lassmann M, Brans B, Flux G. EANM procedure guidelines for 131I-meta-iodobenzylguanidine (131I-mIBG) therapy. Eur J Nucl Med Mol Imaging 2008; 35(5): 1039-47.
[http://dx.doi.org/10.1007/s00259-008-0715-3] [PMID: 18274745]
[111]
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(12): 1889-96.
[http://dx.doi.org/10.1200/JCO.1992.10.12.1889] [PMID: 1453204]
[112]
Kosmin MA, Bomanji JB, Cork NJ, Shankar A, Gaze MN. Hypertension complicating 131I-meta-iodobenzylguanidine therapy for neuroblastoma. Eur J Nucl Med Mol Imaging 2012; 39(4): 597-601.
[http://dx.doi.org/10.1007/s00259-011-2022-7] [PMID: 22215110]
[113]
DuBois SG, Messina J, Maris JM, et al. Hematologic toxicity of high-dose iodine-131-metaiodobenzylguanidine therapy for advanced neuroblastoma. J Clin Oncol 2004; 22(12): 2452-60.
[http://dx.doi.org/10.1200/JCO.2004.08.058] [PMID: 15197208]
[114]
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(3): 500-6.
[http://dx.doi.org/10.1200/JCO.2005.03.6400] [PMID: 16421427]
[115]
Clement SC, van Eck-Smit BL, van Trotsenburg AS, Kremer LC, Tytgat GA, van Santen HM. Long-term follow-up of the thyroid gland after treatment with 131I-Metaiodobenzylguanidine in children with neuroblastoma: importance of continuous surveillance. Pediatr Blood Cancer 2013; 60(11): 1833-8.
[http://dx.doi.org/10.1002/pbc.24681] [PMID: 23832530]
[116]
Hartmann O, Valteau-Couanet D, Vassal G, Lapierre V, Brugières L, Delgado R, et al. Prognostic factors in metastatic neuroblastoma in patients over 1 year of age treated with high-dose chemotherapy and stem cell transplantation: A multivariate analysis in 218 patients treated in a single institution. Bone Marrow Transplant 1999.
[117]
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; 47(1): 31-40.
[PMID: 12714952]
[118]
Park JR, Bagatell R, London WB, et al. Children’s Oncology Group’s 2013 blueprint for research: neuroblastoma. Pediatr Blood Cancer 2013; 60(6): 985-93.
[http://dx.doi.org/10.1002/pbc.24433] [PMID: 23255319]
[119]
Cotterill SJ, Parker L, More L, Craft AW. Neuroblastoma: changing incidence and survival in young people aged 0-24 years. A report from the North of England Young Persons’ Malignant Disease Registry. Med Pediatr Oncol 2001; 36(1): 231-4.
[http://dx.doi.org/10.1002/1096-911X(20010101)36:1<231::AID-MPO1056>3.0.CO;2-U] [PMID: 11464892]
[120]
Schor NF. Neuroblastoma as a neurobiological disease. J Neurooncol 1999; 41(2): 159-66.
[http://dx.doi.org/10.1023/A:1006171406740] [PMID: 10222436]
[121]
Gautschi O, Heighway J, Mack PC, Purnell PR, Lara PN Jr, Gandara DR. Aurora kinases as anticancer drug targets. Clin Cancer Res 2008; 14(6): 1639-48.
[http://dx.doi.org/10.1158/1078-0432.CCR-07-2179] [PMID: 18347165]
[122]
Donfrancesco A, De Ioris MA, McDowell HP, et al. Gefitinib in combination with oral topotecan and cyclophosphamide in relapsed neuroblastoma: pharmacological rationale and clinical response. Pediatr Blood Cancer 2010; 54(1): 55-61.
[http://dx.doi.org/10.1002/pbc.22219] [PMID: 19821523]
[123]
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(2): 254-8.
[http://dx.doi.org/10.1002/pbc.21132] [PMID: 17262795]
[124]
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(5): 1227-34.
[http://dx.doi.org/10.1002/ijc.24069] [PMID: 19058199]
[125]
Carboni JM, Wittman M, Yang Z, et al. BMS-754807, a small molecule inhibitor of insulin-like growth factor-1R/IR. Mol Cancer Ther 2009; 8(12): 3341-9.
[http://dx.doi.org/10.1158/1535-7163.MCT-09-0499] [PMID: 19996272]
[126]
Kang J, Rychahou PG, Ishola TA, Mourot JM, Evers BM, Chung DH. N-myc is a novel regulator of PI3K-mediated VEGF expression in neuroblastoma. Oncogene 2008; 27(28): 3999-4007.
[http://dx.doi.org/10.1038/onc.2008.15] [PMID: 18278068]
[127]
Sims TL, Williams RF, Ng CY, Rosati SF, Spence Y, Davidoff AM. Bevacizumab suppresses neuroblastoma progression in the setting of minimal disease. Surgery 2008; 144(2): 269-75.
[http://dx.doi.org/10.1016/j.surg.2008.04.009] [PMID: 18656635]
[128]
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(2): 147-52.
[http://dx.doi.org/10.1002/ijc.11187] [PMID: 12800187]
[129]
Calafiore L, Amoroso L, Della Casa Alberighi O, et al. Two-stage phase II study of imatinib mesylate in subjects with refractory or relapsing neuroblastoma. Ann Oncol 2013; 24(5): 1406-13.
[http://dx.doi.org/10.1093/annonc/mds648] [PMID: 23396605]
[130]
Maurer BJ, Kang MH, Villablanca JG, et al. Phase I trial of fenretinide delivered orally in a novel organized lipid complex in patients with relapsed/refractory neuroblastoma: a report from the New Approaches to Neuroblastoma Therapy (NANT) consortium. Pediatr Blood Cancer 2013; 60(11): 1801-8.
[http://dx.doi.org/10.1002/pbc.24643] [PMID: 23813912]
[131]
Gurunathan S, Kang MH, Qasim M, Kim JH. Nanoparticle-mediated combination therapy: Two-in-one approach for cancer. Int J Mol Sci 2018; 19(10): 3264.
[http://dx.doi.org/10.3390/ijms19103264] [PMID: 30347840]
[132]
de Kraker J, Hoefnagel KA, Verschuur AC, van Eck B, van Santen HM, Caron HN. Iodine-131-metaiodobenzylguanidine as initial induction therapy in stage 4 neuroblastoma patients over 1 year of age. Eur J Cancer 2008; 44(4): 551-6.
[http://dx.doi.org/10.1016/j.ejca.2008.01.010] [PMID: 18267358]
[133]
Schmidt M, Simon T, Hero B, Dietlein M, Sudbrock F, Bongartz R, et al. Is there a benefit of 131I-MIBG therapy in the treatment of children with stage 4 neuroblastoma? A retrospective evaluation of the German Neuroblastoma Trial NB97 and implications for The German Neuroblastoma Trial NB2004. Nucl Med (Stuttg) 45(4): 145-51. [PMID: 16964339].
[134]
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(7): 1124-9.
[http://dx.doi.org/10.1002/pbc.23062] [PMID: 21495159]
[135]
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(7): 1020-5.
[http://dx.doi.org/10.1200/JCO.2007.15.7628] [PMID: 19171714]
[136]
Mastrangelo R, Tornesello A, Riccardi R, et al. A new approach in the treatment of stage IV neuroblastoma using a combination of I131 meta-iodobenzylguanidine (MIBG) and cisplatin European Journal of Cancer 1995; 31(4): 606-11.
[137]
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(8): 2142-9.
[http://dx.doi.org/10.1200/JCO.2002.08.124] [PMID: 11956276]
[138]
Mullassery D, Dominici C, Jesudason EC, McDowell HP, Losty PD. Neuroblastoma: contemporary management. Arch Dis Child Educ Pract Ed 2009; 94(6): 177-85.
[http://dx.doi.org/10.1136/adc.2008.143909] [PMID: 19933390]
[139]
Rybak L, Mukherjea D, Jajoo S, Ramkumar V. Cisplatin ototoxicity and protection: Clinical and experimental studies. Tohoku J Exp Med 2009; 219(3): 177-86.
[140]
Killock D. Sodium thiosulfate halves the risk of cisplatin-induced hearing loss. Nat Rev Clin Oncol 2018; 15(9): 533.
[http://dx.doi.org/10.1038/s41571-018-0067-2] [PMID: 29980744]
[141]
Elzembely MM, Park JR, Riad KF, et al. Acute Complications after high-dose chemotherapy and stem-cell rescue in pediatric patients with high-risk neuroblastoma treated in countries with different resources JGO . 2018.
[http://dx.doi.org/10.1200/JGO.17.00118]
[142]
Wood J, Ver Halen J, Samant S, Florendo N. Radiation-induced sarcoma masquerading as osteoradionecrosis: case report and literature review. J Laryngol Otol 2015; 129(3): 279-82.
[http://dx.doi.org/10.1017/S0022215114003326] [PMID: 25650528]
[143]
Mitchell MJ, Logan PM. Radiation-induced changes in bone. Radiographics 1998; 18(5): 1125-36.
[http://dx.doi.org/10.1148/radiographics.18.5.9747611] [PMID: 9747611]
[144]
Landier W, Armenian S, Bhatia S. Late effects of childhood cancer and its treatment. Pediatr Clin North Am 2015; 62(1): 275-300.
[http://dx.doi.org/10.1016/j.pcl.2014.09.017] [PMID: 25435123]
[145]
Kushner BH, Kramer K, Cheung NK. Chronic neuroblastoma. Cancer 2002; 95(6): 1366-75.
[http://dx.doi.org/10.1002/cncr.10800] [PMID: 12216106]
[146]
Abbas AA, Khalid KA. Chronic persistent neuroblastoma. A unique presentation and clinical course in a six-year-old child. A case report. Clin in Oncol J 2019; 4(1677): 1-6.

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