Advances in treatment for
children with high-risk
Over the last 25 years, research has dramatically improved the way high-risk neuroblastoma is treated.
A study (CCG-3891) conducted between 1991 and 1996, evaluated the addition of autologous stem cell transplant to consolidation treatment in high-risk neuroblastoma patients. All patients received the same induction chemotherapy and had surgical resection of their tumor. They were then randomized to receive a consolidation regimen with additional high-dose chemotherapy (myeloablative), total body irradiation, and autologous stem cell transplant or to receive a consolidation regimen of three additional cycles of standard chemotherapy without radiation or stem cell transplant. The 5-year event-free survival (EFS) for patients randomized to the autologous stem cell transplant arm was 30%, compared to 19% in those randomized to chemotherapy alone. Of note, some patients in this study (CCG-3891) went on to receive post-consolidation isotretinoin (cis-retinoic acid).
An additional study (ANBL0032) was done from 2001 to 2009, by the Children's Oncology Group (COG), to evaluate post-consolidation therapy in high-risk neuroblastoma patients. In this study, patients received standard induction chemotherapy, consolidation therapy with stem cell transplant and radiation, and were then randomized to receive post-consolidation treatment with isotretinoin (cis-retinoic acid) alone or to receive isotretinoin plus antibody therapy with dinutuximab (Unituxin), granulocyte-macrophage colony-stimulating factor (GMCSF), and interleukin 2 (IL-2). The 2-year event-free survival for patients randomized to the antibody therapy arm was 66%, compared to 46% in those that received isotretinoin (cis-retinoic acid) alone.
Event-free survival (EFS): A medical research term that defines the amount of time a patient lives without the disease, the disease getting worse, a new cancer developing, or the patient dying while being treated.
clinical trial study