Abstract
OBJECTIVES: Neuroblastoma (NB) is a common and life-threatening pediatric solid tumor with a poor prognosis, especially in high-risk patients. Although anti-disialoganglioside GD2 (GD2) monoclonal antibodies improve survival, relapse and resistance remain major challenges. This study aimed to evaluate the safety and efficacy of GD2-targeted chimeric antigen receptor (CAR)-T-cell therapy in NB patients. METHODS: A literature search was conducted in PubMed, Embase, Scopus, and Web of Science until September 30, 2025. Eligible studies were clinical trials that evaluated the efficacy and safety of GD2-targeted CAR-T-cell therapy in NB patients. Two reviewers independently performed study selection, data extraction, and risk-of-bias assessment. The random-effects meta-analysis model was used to calculate pooled amounts. RESULTS: Eight studies that included 146 patients with NB were included. The pooled complete response (CR) rate was 39.57% (21.17-57.96), and the partial response (PR) rate was 15.83% (5.02-30.45). Additionally, the rates of progressive disease (PD) and stable disease (SD) were 20.9% (3.06-46.67) and 30.76% (12.81-51.91), respectively. The most common "any grade" adverse events (AEs) was anemia, at 97.43% (81.51-100), and the most common "grade ≥ 3" AEs was neutropenia, at 93.46% (72.65-100). Subgroup analyses revealed that CAR-T-cell generation and its components influenced efficacy and safety. CONCLUSION: CAR-T-cell therapy targeting the GD2 antigen is promising for treating NB. However, its efficacy is moderate, and treatment can lead to hematologic toxicities such as anemia, neutropenia, and thrombocytopenia, which require careful monitoring.