Bevacizumab, Irinotecan, or Topotecan Added to Temozolomide for Children With Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON‑Neuroblastoma Trial
In the BEACON Neuroblastoma trial, the addition of bevacizumab met protocol‑defined success criteria for overall response rate (ORR) and appeared to improve progression‑free survival (PFS).
Outcomes for children with relapsed and refractory high‑risk neuroblastoma (RR‑HRNB) remain dismal. The BEACON Neuroblastoma trial (EudraCT 2012‑000072‑42) evaluated three backbone chemotherapy regimens and the addition of the anti‑angiogenic agent bevacizumab (B). Patients age 1‑21 years with RR‑HRNB with adequate organ function and performance status were randomly assigned in a 3×2 factorial design to temozolomide (T), irinotecan‑temozolomide (IT), or topotecan‑temozolomide (TTo) with or without B. The primary end point was best overall response (complete or partial) rate (ORR) during the first six courses, by RECIST or International Neuroblastoma Response Criteria for patients with measurable or evaluable disease, respectively. Safety, progression‑free survival (PFS), and overall survival (OS) time were secondary end points.
This randomized phase II study demonstrates that adding bevacizumab to standard temozolomide‑based regimens can increase the proportion of children with relapsed or refractory high‑risk neuroblastoma who achieve a complete or partial response and may prolong progression‑free survival, providing a new therapeutic strategy for this difficult‑to‑treat disease.
Evidence level: Számítógépes vagy elméleti. Modellből vagy adatbányászatból származó jel.
Related signals
- Targeted immunotherapies for anaplastic lymphoma kinase-positive pediatric tumors: current advances and future perspectives
- Coexpression of MYCN and ALK Induces Neuroblastoma-Like Tumors From Human iPS Cell-Derived Cranial Neural Crest Cells.
- Frequency and Clinical Significance of Clonal and Subclonal Driver Mutations in High‑Risk Neuroblastoma at Diagnosis: A Children's Oncology Group Study
- Treatment and Survival of Childhood Neuroblastoma: Evidence from a Population-Based Study in the United States
- Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study