Tislelizumab combined with Anti-GD2 antibody-based chemoimmunotherapy for pediatric relapsed/refractory high-risk neuroblastoma: a two-Case Report.
AI interpretation is pending for this paper.
Open original publication →What the AI sees
Not AI summarized yet.
Research significance
Pending deeper interpretation.
Source abstract
Neuroblastoma (NB) is one of the most common extracranial solid tumors in children. Patients with high-risk metastatic disease remain at substantial risk of relapse and have poor long-term outcomes. No standard salvage regimen has been established for relapsed/refractory (R/R) NB, and the role of immune checkpoint inhibitors in pediatric NB remains investigational. Here, we report two pediatric patients with R/R high-risk NB who received a tislelizumab-containing multi-agent regimen consisting of tislelizumab, an anti-GD2 monoclonal antibody, GM-CSF support, and sequential mICE/VIT chemotherapy. The anti-GD2 antibody, dinutuximab beta or naxitamab, was selected according to each patient's insurance coverage. Both patients tolerated treatment well, and no severe immune-related adverse events were observed during the reported follow-up period. After four cycles of combination therapy, Patient 1 showed marked regression of soft-tissue lesions and clearance of bone marrow involvement. Patient 2 achieved a complete response and remained disease-free during the 5-month follow-up period after treatment completion. However, the durability of this response remains uncertain because of the limited follow-up duration. These preliminary short-term observations suggest that this multi-agent regimen may be a feasible salvage approach for selected pediatric patients with R/R high-risk NB. However, the durability of these antitumor responses cannot be confirmed given the limited surveillance period. Notably, this retrospective two-case observation cannot distinguish the independent antitumor contribution of tislelizumab alone, as multiple therapeutic components were administered simultaneously without control groups to isolate single-agent efficacy. Larger prospective studies are warranted to further evaluate the efficacy and safety of this approach.