The overall survival for children with high risk NBL is roughly 20-35%.
The disease is generally very responsive to chemotherapy, but there is a high relapse rate.
Four components of treatment:
- Induction - Aggressive multiagent chemotherapy (cyclophosphamide, topotecan, cisplatin, etoposide, doxorubicin, vincristine).
- Local Control - surgery and radiation therapy (RT).
- Consolidation - autologous bone marrow transplant.
- Biologic agents - isotretinoin (cis-RA)
COG-ANBL0532: To improve event free survival (EFS), this protocol uses:
- Dose intensification of the induction chemotherapy (with addition of dose-intensive topotecan and cyclophosphamide in induction)
- Randomized substitution of two cycles of topotecan and cyclophosphamide for two cycles of vincristine, cyclophosphamide, and doxorubicin to assess if this improves outcome.
- Tandem transplant using marrow support
- Randomization between two cycles of myeloablative chemotherapy and stem cell transplantation vs a single cycle of myeloblative chemotherapy and stem cell transplantation.
- Escalation of RT dose to improve local control
COG-ANBL0032 and COG-ANBL0931: This is a COG non-randomized study looking at the efficacy of monoclonal antibody therapy with granulocyte-macrophage colony-stimulating factor and interleukin-2 combined with cis-retinoic acid following completion of chemotherapy
The New Approaches to Neuroblastoma Therapy (NANT) consortium is studying inclusion of myelobablative doses of iodine-131-meta-iodobenzylguanidine (MIBG) with myeloablative chemotherapy prior to stem cell transplantation in patients with an incomplete response to induction chemotherapy.
External Link:
Treatment of high risk neuroblastoma at the National Cancer Institute
Anti-GD2 Antibody with GM-CSF, Interleukin-2 and Isotretinoin for Neuroblastoma
Yu,A. et al.
