High-Risk Neuroblastoma Treatment Review

Valeria Smith, Jennifer Foster, Valeria Smith, Jennifer Foster

Abstract

Neuroblastoma is the most common extracranial solid tumor in children. One subset, high-risk neuroblastoma, is very difficult to treat and requires multi-modal therapy. Intensification of therapy has vastly improved survival rates, and research is focused on novel treatments to further improve survival rates. The current treatment schema is divided into three stages-induction, consolidation, and maintenance. This review serves as an overview of the current treatment for high-risk neuroblastoma and a glimpse at current research for future therapy.

Keywords: diagnosis; high-risk neuroblastoma; neuroblastoma; treatment.

Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
High-risk neuroblastoma treatment overview.

References

    1. Brodeur G., Hogarty M., Bagatell R., Mosse Y., Maris J. Neuroblastoma. In: Pizzo P., Poplack D., editors. Principles and Practice of Pediatric Oncology. 7th ed. Wolters Kluwer; Philadelphia, PA, USA: 2016. pp. 772–798.
    1. Cohn S.L., Pearson A.D.J., London W.B., Monclair T., Ambros P.F., Brodeur G.M., Faldum A., Hero B., Iehara T., Machin D., et al. The International Neuroblastoma Risk Group (INRG) Classification System: An INRG Task Force Report. J. Clin. Oncol. 2009;27:289–297. doi: 10.1200/JCO.2008.16.6785.
    1. Monclair T., Brodeur G.M., Ambros P.F., Brisse H.J., Cecchetto G., Holmes K., Kaneko M., London W.B., Matthay K.K., Nuchtern J.G., et al. The International Neuroblastoma Risk Group (INRG) Staging System: An INRG Task Force Report. J. Clin. Oncol. 2009;27:298–303. doi: 10.1200/JCO.2008.16.6876.
    1. Pinto N.R., Applebaum M.A., Volchenboum S.L., Matthay K.K., London W.B., Ambros P.F., Nakagawara A., Berthold F., Schleiermacher G., Park J.R., et al. Advances in Risk Classification and Treatment Strategies for Neuroblastoma. J. Clin. Oncol. 2015;33:3008–3017. doi: 10.1200/JCO.2014.59.4648.
    1. Louis C.U., Shohet J.M. Neuroblastoma: Molecular Pathogenesis and Therapy. Annu. Rev. Med. 2015;66:49–63. doi: 10.1146/annurev-med-011514-023121.
    1. Peinemann F., Kahangire D.A., van Dalen E.C., Berthold F. Rapid COJEC versus standard induction therapies for high-risk neuroblastoma. Cochrane Database Syst. Rev. 2015;60:1135–1151. doi: 10.1002/14651858.CD010774.pub2.
    1. Kushner B.H., Kramer K., LaQuaglia M.P., Modak S., Yataghene K., Cheung N.-K.V. Reduction From Seven to Five Cycles of Intensive Induction Chemotherapy in Children With High-Risk Neuroblastoma. J. Clin. Oncol. 2004;22:4888–4892. doi: 10.1200/JCO.2004.02.101.
    1. Garaventa A., Poetschger U., Valteau-Couanet D., Castel V., Elliott M., Ash S., Chan G.C., Laureys G., Beck Popovic M., Vettenranta K., et al. The Randomised Induction for High-Risk Neuroblastoma Comparing COJEC and the N5-MSKCC Regimen: Early Results from the HR-NBL1.5/SIOPEN Trial; Proceedings of the Advances in Neuroblastoma Research Association (ANARA); San Francisco, CA, USA. 9–12 May 2018.
    1. Kreissman S.G., Seeger R.C., Matthay K.K., London W.B., Sposto R., Grupp S.A., Haas-Kogan D.A., LaQuaglia M.P., Alice L.Y., Diller L., 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;14:999–1008. doi: 10.1016/S1470-2045(13)70309-7.
    1. Adkins E.S., Sawin R., Gerbing R.B., London W.B., Matthay K.K., Haase G.M. Efficacy of complete resection for high-risk neuroblastoma: A children’s cancer group study. J. Pediatr. Surg. 2004;39:931–936. doi: 10.1016/j.jpedsurg.2004.02.041.
    1. Simon T., Häberle B., Hero B., von Schweinitz D., Berthold F. Role of Surgery in the Treatment of Patients With Stage 4 Neuroblastoma Age 18 Months or Older at Diagnosis. J. Clin. Oncol. 2013;31:752–758. doi: 10.1200/JCO.2012.45.9339.
    1. Fischer J., Pohl A., Volland R., Hero B., Dübbers M., Cernaianu G., Berthold F., Schweinitz D., Simon T. Complete surgical resection improves outcome in INRG high-risk patients with localized neuroblastoma older than 18 months. BMC Cancer. 2017;17:520. doi: 10.1186/s12885-017-3493-0.
    1. Matthay K.K., Villablanca J.G., Seeger R.C., Stram D.O., Harris R.E., Ramsay N.K., Swift P., Shimada H., Black C.T., Brodeur G.M., 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;341:1165–1173. doi: 10.1056/NEJM199910143411601.
    1. Matthay K.K., Reynolds C.P., Seeger R.C., Shimada H., Adkins E.S., Haas-Kogan D., Gerbing R.B., London W.B., Villablanca J.G. 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. doi: 10.1200/JCO.2007.13.8925.
    1. Park J.R., Kreissman S.G., London W.B., Naranjo A., Cohn S.L., Hogarty M.D., Tenney S.C., Haas-Kogan D., Shaw P.J., Geiger J.D., et al. A phase III randomized clinical trial (RCT) of tandem myeloablative autologous stem cell transplant (ASCT) using peripheral blood stem cell (PBSC) as consolidation therapy for high-risk neuroblastoma (HR-NB): A Children’s Oncology Group (COG) study; Proceedings of the American Society of Clinical Oncology (ASCO) Conference; Chicago, IL, USA. 3–7 June 2016.
    1. Qayed M., Chiang K.-Y., Ricketts R., Alazraki A., Tahvildari A., Haight A., George B., Esiashvili N., Katzenstein H.M. Tandem stem cell rescue as consolidation therapy for high-risk neuroblastoma. Pediatr. Blood Cancer. 2011;58:448–452. doi: 10.1002/pbc.23155.
    1. Elborai Y., Hafez H., Moussa E.A., Hammad M., Hussein H., Lehmann L., Elhaddad A. Comparison of toxicity following different conditioning regimens (busulfan/melphalan and carboplatin/etoposide/melphalan) for advanced stage neuroblastoma: Experience of two transplant centers. Pediatr. Transplant. 2015;20:284–289. doi: 10.1111/petr.12638.
    1. Ladenstein R., Pötschger U., Pearson A.D.J., Brock P., Luksch R., Castel V., Yaniv I., Papadakis V., Laureys G., Malis J., et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): An international, randomised, multi-arm, open-label, phase 3 trial. Lancet Oncol. 2017;18:500–514. doi: 10.1016/S1470-2045(17)30070-0.
    1. Gaze M.N., Boterberg T., Dieckmann K., Hörmann M., Gains J.E., Sullivan K.P., Ladenstein R. Results of a Quality Assurance Review of External Beam Radiation Therapy in the International Society of Paediatric Oncology (Europe) Neuroblastoma Group’s High-risk Neuroblastoma Trial: A SIOPEN Study. Radiat. Oncol. Boil. 2013;85:170–174. doi: 10.1016/j.ijrobp.2012.05.004.
    1. Mazloom A., Louis C.U., Nuchtern J., Kim E., Russell H., Allen-Rhoades W., Krance R., Paulino A.C. Radiation Therapy to the Primary and Postinduction Chemotherapy MIBG-Avid Sites in High-Risk Neuroblastoma. Int. J. Radiat. Oncol. Boil. Phys. 2014;90:858–862. doi: 10.1016/j.ijrobp.2014.07.019.
    1. Casey D.L., Kushner B.H., Cheung N.-K.V., Modak S., LaQuaglia M.P., Wolden S.L. Local Control with 21-Gy Radiation Therapy for High-Risk Neuroblastoma. Int. J. Radiat. Oncol. Boil. Phys. 2016;96:393–400. doi: 10.1016/j.ijrobp.2016.05.020.
    1. Brodeur G.M., Pritchard J., Berthold F., Carlsen N.L., Castel V., Castelberry R.P., De Bernardi B., Evans A.E., Favrot M., Hedborg F. Revisions of the international criteria for neuroblastoma diagnosis, staging and response to treatment. Prog. Clin. Biol. Res. 1994;385:363–369. doi: 10.1200/JCO.1993.11.8.1466.
    1. Hill-Kayser C., Tochner Z., Both S., Lustig R., Reilly A., Balamuth N., Womer R., Maris J., Grupp S., Bagatell R. Proton versus photon radiation therapy for patients with high-risk neuroblastoma: The need for a customized approach. Pediatr. Blood Cancer. 2013;60:1606–1611. doi: 10.1002/pbc.24606.
    1. Hattangadi J.A., Rombi B., Yock T.I., Broussard G., Friedmann A.M., Huang M., Chen Y.L., Lu H.M., Kooy H., MacDonald S.M. Proton Radiotherapy for High-Risk Pediatric Neuroblastoma: Early Outcomes and Dose Comparison. Int. J. Radiat. Oncol. Boil. Phys. 2012;83:1015–1022. doi: 10.1016/j.ijrobp.2011.08.035.
    1. Peinemann F., van Dalen E.C., Tushabe D.A., Berthold F. Retinoic acid post consolidation therapy for high-risk neuroblastoma patients treated with autologous hematopoietic stem cell transplantation. Cochrane Database Syst. Rev. 2015;1:CD010685.
    1. Yu A.L., Gilman A.L., Ozkaynak M.F., London W.B., Kreissman S.G., Chen H.X., Smith M., Anderson B., Villablanca J.G., Matthay K.K., et al. Anti-GD2 Antibody with GM-CSF, Interleukin-2, and Isotretinoin for Neuroblastoma. N. Engl. J. Med. 2010;363:1324–1334. doi: 10.1056/NEJMoa0911123.
    1. Cheung N.-K.V., Cheung I.Y., Kushner B.H., Ostrovnaya I., Chamberlain E., Kramer K., Modak S. Murine Anti-GD2 Monoclonal Antibody 3F8 Combined With Granulocyte-Macrophage 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. doi: 10.1200/JCO.2011.41.3807.
    1. Zhou M.J., Doral M.Y., DuBois S.G., Villablanca J.G., Yanik G.A., Matthay K.K. Different outcomes for relapsed vs. refractory neuroblastoma after therapy with 131I-metaiodobenzylguanidine (131I-MIBG) Eur. J. Cancer. 2015;51:2465–2472. doi: 10.1016/j.ejca.2015.07.023.
    1. Pugh T.J., Morozova O., Attiyeh E.F., Asgharzadeh S., Wei J.S., Auclair D., Carter S.L., Cibulskis K., Hanna M., Kiezun A., et al. The genetic landscape of high-risk neuroblastoma. Nat. Genet. 2013;45:279–284. doi: 10.1038/ng.2529.
    1. Peifer M., Hertwig F., Roels F., Dreidax D., Gartlgruber M., Menon R., Krämer A., Roncaioli J.L., Sand F., Heuckmann J.M. Telomerase activation by genomic rearrangements in high-risk neuroblastoma. Nature. 2015;526:700–704. doi: 10.1038/nature14980.
    1. Depuydt P., Boeva V., Hocking T.D., Cannoodt R., Ambros I.M., Ambros P.F., Asgharzadeh S., Attiyeh E.F., Combaret V., Defferrari R., et al. Genomic Amplifications and Distal 6q Loss: Novel Markers for Poor Survival in High-risk Neuroblastoma Patients. JNCI J. Natl. Cancer Inst. :2018. doi: 10.1093/jnci/djy022.
    1. Lee J.W., Lee S., Cho H.W., Ma Y., Yoo K.H., Sung K.W., Koo H.H., Cho E.J., Lee S.K., Lim D.H. Incorporation of high-dose 131I-metaiodobenzylguanidine treatment into tandem high-dose chemotherapy and autologous stem cell transplantation for high-risk neuroblastoma: Results of the SMC NB-2009 study. J. Hematol. Oncol. 2017;10:108. doi: 10.1186/s13045-017-0477-0.
    1. Mastrangelo S., Rufini V., Ruggiero A., Di Giannatale A., Riccardi R. Treatment of advanced neuroblastoma in children over 1 year of age: The critical role of 131I-metaiodobenzylguanidine combined with chemotherapy in a rapid induction regimen. Pediatr. Blood Cancer. 2011;56:1032–1040. doi: 10.1002/pbc.22986.
    1. Krytska K., Ryles H.T., Sano R., Raman P., Infarinato N., Hansel T.D., Makena M.R., Song M.M., Reynolds C.P., Mossé Y.P. Crizotinib Synergizes with Chemotherapy in Preclinical Models of Neuroblastoma. Clin. Cancer Res. 2016;22:948–960. doi: 10.1158/1078-0432.CCR-15-0379.
    1. Mossé Y.P., Lim M.S., Voss S.D., Wilner K., Ruffner K., Laliberte J., Rolland D., Balis F.M., Maris J.M., Weigel B.J., 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 Oncol. 2013;14:472–480. doi: 10.1016/S1470-2045(13)70095-0.
    1. Bassiri H., Benavides A., Haber M., Gilmour S.K., Norris M.D., Hogarty M.D. Translational development of difluoromethylornithine (DFMO) for the treatment of neuroblastoma. Transl. Pediatr. 2015;4:226–238. doi: 10.3978/j.issn.2224-4336.2015.04.06.
    1. Saulnier Sholler G.L., Gerner E.W., Bergendahl G., MacArthur R.B., VanderWerff A., Ashikaga T., Bond J.P., Ferguson W., Roberts W., Wada R.K., et al. A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/Refractory Neuroblastoma. PLoS ONE. 2015;10:e0127246. doi: 10.1371/journal.pone.0127246.
    1. Kushner B.H., Cheung I.Y., Modak S., Kramer K., Ragupathi G., Cheung N.-K.V. Phase I Trial of a Bivalent Gangliosides Vaccine in Combination with β-Glucan for High-Risk Neuroblastoma in Second or Later Remission. Clin. Cancer Res. 2014;20:1375–1382. doi: 10.1158/1078-0432.CCR-13-1012.

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