Abstract
Neuroblastoma is the most common extracranial solid tumor in children, accounting for 15% of all pediatric cancer deaths. High-risk neuroblastoma (HRNB) is a particularly difficult-to-treat form of the disease that requires aggressive multimodality therapy, including induction chemotherapy, consolidation therapy with high-dose chemotherapy and autologous stem cell transplant, and maintenance therapy with dinutuximab beta. Despite treatment advances, the prognosis of these patients remains poor. As a better response to induction therapy has been associated with prolonged survival in patients with HRNB, we hypothesized that early use of dinutuximab beta—post-induction chemotherapy—may improve patient outcomes. We describe here our experience of administering at least one cycle of dinutuximab beta post-induction and prior to surgery in three children with HRNB who did not demonstrate a complete response to induction chemotherapy. All three patients achieved complete remission. Early use of dinutuximab beta may therefore have the potential to improve outcomes in patients with HRNB.
Highlights
Neuroblastoma is a rare malignant disease, it is the most common extracranial solid tumor in childhood, accounting for 15% of all pediatric cancer deaths [1]
Any patient with MYCN amplification is classified as high risk, as are patients with metastatic disease who are ≥18 months of age (International Neuroblastoma Staging System (INSS) Stage 4) and those
It was approved by the European Medicines Agency in 2017 for the treatment of Highrisk neuroblastoma (HRNB) in patients aged ≥12 months who have achieved at least a partial response (PR) to induction chemotherapy and received myeloablative therapy and stem cell transplant and patients with a history of relapsed or refractory neuroblastoma, with or without residual disease [9]
Summary
Neuroblastoma is a rare malignant disease, it is the most common extracranial solid tumor in childhood, accounting for 15% of all pediatric cancer deaths [1]. Dinutuximab beta is a monoclonal antibody that targets disialoganglioside 2 (GD2), which is ubiquitously overexpressed on neuroblastoma cells [8] It was approved by the European Medicines Agency in 2017 for the treatment of HRNB in patients aged ≥12 months who have achieved at least a PR to induction chemotherapy and received myeloablative therapy and stem cell transplant and patients with a history of relapsed or refractory neuroblastoma, with or without residual disease [9]. While recent follow-up data confirmed the long-term survival benefit of dinutuximab in these patients, the magnitude of the benefit had decreased over time due to late relapses [12], indicating the need to identify new treatment approaches to further improve outcomes. We report the outcomes of three patients with HRNB treated with dinutuximab beta after induction therapy and before surgery
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