Abstract
Background
Despite advances in multimodal therapy, high-risk neuroblastoma (HR-NB) continues to have poor outcomes. Response to induction chemotherapy is a key predictor of long-term survival, yet intensive multi-agent regimens have not substantially improved prognosis.Objectives
This study aims to evaluate the efficacy and safety of adding bevacizumab to conventional induction chemotherapy in HR-NB.Methods
This prospective, multicenter, randomized controlled trial will enroll newly diagnosed high-risk neuroblastoma patients, randomized to receive either conventional induction chemotherapy alone (control) or bevacizumab plus conventional induction chemotherapy (intervention). Response to induction therapy will be assessed according to standard criteria, and patients achieving a complete or good partial response will proceed to autologous stem cell transplantation (ASCT). All participants will be followed for at least 2 years to evaluate event-free survival (EFS) and overall survival (OS). The primary outcome, response rate, will be reported as a proportion, and EFS and OS will be analyzed using Kaplan-Meier plots and log-rank tests. The secondary outcomes (toxicity and minimal residual disease) will be reported as proportions. Prognostic factors will be evaluated with univariate and multivariate Cox regression analyses.Expected outcomes
The study aims to demonstrate that adding bevacizumab to conventional induction chemotherapy improves response rates, increases the proportion of patients eligible for autologous stem cell transplantation (ASCT), and improves survival outcomes, with manageable toxicities.Trial registration
This study is registered with the Clinical Trials Registry of India (CTRI/2024/09/074065, dated: 19/09/2024).