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Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of High-Risk Neuroblastoma


Standard Treatment Options

Patients classified as high risk receive treatment with an aggressive regimen of combination chemotherapy consisting of very high drug doses, generally termed induction. Drugs often used include cyclophosphamide, ifosfamide, cisplatin, carboplatin, vincristine, doxorubicin, etoposide, and topotecan. COG has completed a pilot study of induction demonstrating the feasibility of substituting two cycles of topotecan and cyclophosphamide for two cycles of vincristine, cyclophosphamide, and doxorubicin.[16] After a response to chemotherapy, resection of the primary tumor should be attempted, followed by myeloablative chemotherapy and stem cell rescue (i.e., bone marrow and/or peripheral blood stem cell transplantation). Whether or not harvested stem cells should be purged of neuroblastoma cells has been studied in a randomized fashion. There was no advantage to purging.[17] Two or more sequential cycles of myeloablative chemotherapy and stem cell rescue given in a tandem fashion has been studied and feasibility was established.[7,18] It is now under clinical evaluation in COG. Radiation to the primary tumor site should be undertaken whether or not a complete excision was obtained. The optimal dose of radiation therapy has not been determined. Radiation of sites of metastatic disease is determined on an individual case basis. After recovery, patients are treated with oral 13-cis -retinoic acid for 6 months. Both myeloablative therapy and postchemotherapy retinoic acid improve outcome in patients categorized as high risk.[3,5] For high risk-patients in remission following HSCT, compared to retinoic acid alone, chimeric anti-GD2 antibody ch14.18 combined with granulocyte-macrophage colony stimulating factor and interleukin-2 and given in concert with retinoic acid improves EFS.[6]

Treatment Options Under Clinical Evaluation

The following are examples of national and/or institutional clinical trials that are currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.

  • COG-ANBL09P1 (Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing SCT, Radiation Therapy, and Maintenance Therapy With Isotretinoin ): This limited-participation pilot study for children with newly diagnosed high-risk neuroblastoma assesses the tolerability and feasibility of an induction regimen containing five cycles of multi-agent chemotherapy and a block of 131 I-MIBG/irinotecan/vincristine followed by a consolidation regimen of busulfan/melphalan with autologous stem cell rescue and external-beam radiation therapy.
  • COG-ANBL0032 (Isotretinoin With or Without Monoclonal Antibody, Interleukin-2, and Sargramostim Following SCT in Treating Patients With Neuroblastoma): The COG is studying, in a nonrandomized fashion, the use of monoclonal antibody therapy with granulocyte-macrophage colony-stimulating factor and interleukin-2 combined with cis -retinoic acid following chemotherapy.[6,19,20]

Current Clinical Trials

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with neuroblastoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.


WebMD Public Information from the National Cancer Institute

Last Updated: February 25, 2014
This information is not intended to replace the advice of a doctor. Healthwise disclaims any liability for the decisions you make based on this information.
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