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Targeted molecular therapy (modified RIST regimen) in relapsed high risk stage IV neuroblastoma: two cases report

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Submitted   Dec 29, 2017
Published   Jan 2, 2018
Issue    Vol 2 (2020)
DOI   10.25082/TCPP.2018.01.002

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Paolo Indolfi, MD corresponding author
Unit of Pediatric Oncology, Pediatric Department, Second University of Naples, via Luigi De Crecchio 2 80138 Naples, Italy
Selim Corbacioglu, MD
Department of Pediatrics and Adolescent Medicine, University of Regensburg, Regensburg, Germany
Silverio Perrotta, MD
Unit of Pediatric Oncology, Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Francesca Rossi, MD
Unit of Pediatric Oncology, Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Antonio Marte, MD
Department of Pediatric Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Elvira Pota, MD
Unit of Pediatric Oncology,Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Martina Di Martino, MD
Unit of Pediatric Oncology,Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Daniela Di Pinto, MD
Unit of Pediatric Oncology,Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Cristiana Indolfi, MD
Unit of Pediatric Oncology,Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy
Fiorina Casale, MD
Unit of Pediatric Oncology,Department of Women-Children and General and Specialistic Surgery, University of Campania “ Luigi Vanvitelli”, Naples, Italy

Abstract

The prognosis for children with recurrent or refractory neuroblastoma remains a significant clinical challenge, and currently there are no known curative salvage regimens. In this paper we investigated the effect of imatinib with rapamycin and the chemotherapeutic agents temozolomide and irinotecan. We treated two children with recurrent neuroblastoma with this so called RIST protocol. Both patients, off therapy for 15 and 31 months, respectively are well, and developing normally, without any complications. These findings suggest that a combination regimen of RIST may provide a therapeutic benefit with a favorable toxicity profile to a unfortunate subset of patients with neuroblastoma.

Keywords
RIST therapy, neuroblastoma, pediatric cancer

Article Details

How to Cite
Indolfi, P., Corbacioglu, S., Perrotta, S., Rossi, F., Marte, A., Pota, E., Martino, M., Pinto, D., Indolfi, C., & Casale, F. (2018). Targeted molecular therapy (modified RIST regimen) in relapsed high risk stage IV neuroblastoma: two cases report. Theory and Clinical Practice in Pediatrics, 2(1), 29-31. https://doi.org/10.25082/TCPP.2018.01.002
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This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

References

  1. Pourquier P, Waltman JL, Urasaki Y, et al. Topoisomerase-I-mediated cytotoxicity of N-methyl-N- mitro-N-mitrosoguanide: trapping of topoisomerase I by the O6- methylguanine Cancer Res 2001; 61:53-58
  2. Reardon DA, Quinn JA, Rich JN, et al. Phase I trial of irinotecan plus temozolomide in adults with recurrent malignant glioma. Cancer 2005; 104: 1478-1486 https://doi.org/10.1002/cncr.21316
  3. Corbacioglu S, Kilic M, Westhoff MA, et al. Newly identified c-KIT receptor tyrosine kinase ITD in childhood AML induces ligand independent growth and is responsive to a synergistic effects of imatinib and rapamycin. Blood 2006; 108: 3504-3513 https://doi.org/10.1182/blood-2006-05-021691
  4. Corbacioglu S, Steinbach D, Lode HN, et al. The RIST design: a molecularly targeted multimodal approach for the treatment of patients with relapsed and refractory neuroblastoma. J Clin Oncol 2013; 31 (suppl; abstr 10017)
  5. Wagner LM, Crews KR, Iacono LC, et al. Phase I trial of temozolomide and protracted irinotecan in pediatric patients with refractory solid tumors. Clin Cancer Res 2004; 10: 840-848 https://doi.org/10.1158/1078-0432.CCR-03-0175
  6. Nicholson HS, Krailo M, Ames MM, et al. Phase I study of temozolomide in children and adolescents with recurrent solid tumors: a report from the Children's Cancer Group. J Clin Oncol 1998; 16: 3037-3043 https://doi.org/10.1200/JCO.1998.16.9.3037
  7. Estlin EJ, Lashford L, Ablett S, et al. Phase I study of temozolomide in pediatric patients with advanced cancer. United Kingdom Children's Cancer Study Group. Br J Cancer 1998; 78: 652-661 https://doi.org/10.1038/bjc.1998.555
  8. De Sio L, Milano GM, Castellano A, et al. Temozolomide in resistant or relapsed pediatric solid tumors. Pediatr Blood Cancer 2006; 47: 30-36 https://doi.org/10.1002/pbc.20516
  9. 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
  10. Houghton PJ, Stewart CF, Cheshire PJ, et al. Antitumor activity of temozolomide combined with irinotecan is partly independent of 06- methylguanine-DNA methyltransferase and mismatch repair phenotypes in xenograft models. Clin Cancer Res 2000; 6: 4110-4118
  11. Mixon BA, Eckirich MJ, Lowas S, et al. Vincristine, Irinotecan, and Temozolomide for treatment of relapsed alveolar rhabdomyosarcoma. J Pediatr Hematol Oncol 2013; 35: 163-166 https://doi.org/10.1097/MPH.0b013e31825802c2
  12. Beppu K, Jaboine J, Merchant MS, et al. Effect of imatinib mesylate on neuroblastoma tumorigenesis and vascular endothelial growth factor expression. J Natl Cancer Inst 2004; 96: 46-55 https://doi.org/10.1093/jnci/djh004
  13. Vitale 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: 147-152 https://doi.org/10.1002/ijc.11187
  14. Heimberg AB, Wang E, McGary EC, et al. Mechanisms of action of rapamycin in gliomas. Neuro Oncol 2005; 7: 1-11 https://doi.org/10.1215/S1152851704000420
  15. Qayum N, Im J, Stratford MR, et al. Modulation of the tumor microvasculature by phosphoinositide-3 kinase inhibition increases doxorubicin delivery in vivo. Clin cancer Res 2012; 18: 161-169 https://doi.org/10.1158/1078-0432.CCR-11-1413
  16. Saito K, Matsumoto S, Yasui H, et al. Longitudinal imaging studies of tumor microenvironment in mice treated with the mTOR inhibitor rapamycin Plos One 2012; 7: e49456 https://doi.org/10.1371/journal.pone.0049456