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Original Research

Program: Section on Advances in Therapeutics and Technology

P4.A012: Survival Outcomes in Children with High-Risk Neuroblastoma Treated with Anti-GD2 Immunotherapy: A Single-Arm Systematic Review and Meta-Analysis

Monday, September 30, 2024
12:00 PM – 1:00 PM EDT
Location: Poster Hall: Hyatt Regency Orlando, Plaza International Ballroom
Poster Presentations
Background: High-risk Neuroblastoma presents a clinical challenge due to its poor prognosis, with some studies showing a 43% 5-year survival rate in newly diagnosed cases and a 21% 1-year event-free survival rate in relapsed patients. Immunotherapy targeting anti-disialoganglioside (anti-GD2) has emerged as a promising strategy, with agents like Dinutuximab and Naxitamab showing the potential to improve survival outcomes. This meta-analysis aims to assess the impact of anti-GD2 drugs on survival rates in patients with High-risk Neuroblastoma, offering valuable insights into their therapeutic efficacy.

Methods: We systematically searched Pubmed, Cochrane, and EMBASE for clinical trials and prospective cohorts between 2004 and 2023 utilizing anti-GD2 drugs in patients with high-risk neuroblastoma. We pooled the prevalence and the 95% confidence intervals (CI) for the outcomes of interest: overall survival (OS), event-free survival (EFS), progression-free survival (PFS), and objective response rate (ORR). Heterogeneity was assessed using I² statistics. A random-effects model was used for all outcomes. Statistical analyses were performed using R software version 4.3.2.

Results: We included 18 studies comprising 3139 patients. Of these, 11 studies evaluated Dinutuximab, three studies evaluated Naxitamab, two studies hu14.18K322A, and two studies 3F8. Among the included patients, 529 (16.8%) had relapsed or refractory disease, with ages ranging from 1 to 21 years. In a pooled analysis, at one year, the mean EFS stood at 70.47% (95% CI 49.18 to 91.76), while OS reached 92.44% (95% CI 88.34 to 96.56). By the third year, EFS was 55.46% (95% CI 44.80 to 62.14), accompanied by an OS rate of 73.14% (95% CI 69.55 to 76.73). The 5-year EFS held at 47.65% (95% CI 39.07 to 56.25), with OS at 62.12% (95% CI 56.76 to 69.49).

Conclusion: This single-arm meta-analysis suggests that high-risk Neuroblastoma patients may benefit from anti-GD2 immunotherapy, with promising impacts on OS, PFS, and EFS.