Efficacy and Safety of Anti-GD2 Monoclonal Antibodies Combined with GM-CSF in Relapsed and Refractory Neuroblastoma: A Systematic Review and Meta-analysis
Muaz Ahmed1, Anas Nasir2, Dania Hussain3, Suleman Saeed4, Hamid Bin Tariq4, Izma Kashif4, Zoha Kashif4, Tooba Ali4, Maham Amer4, Muhammad Ali Bhatti5, Abdul Rehman6, Zainab Tanveer4, Jaweria Sharif7, Awais Akhtar8, Muhammad Owais Mazhar8, Muhammad Ali Bin Jabir8
1Akhtar Saeed Medical & Dental College, Lahore, Pakistan, 2Sheikh Zayed Medical College, Rahim Yar Khan, Pakistan, 3United Medical And Dental College, Karachi, Pakistan, 4University College of Medicine and Dentistry, Lahore, Pakistan, 5Poonch Medical College Rawalakot, Rawalakot, AJK, Pakistan, 6Services Institute of Medical Sciences, Lahore, Pakistan, 7Allama Iqbal Medical College, Lahore, Pakistan, 8Foundation University Medical College, Islamabad, Pakistan
Objective:
To conduct a systematic review and meta-analysis evaluating the efficacy and safety of anti-GD2 monoclonal antibody therapy combined with GM-CSF in patients with neuroblastoma, with the aim of clarifying its therapeutic value.
Background:
Neuroblastoma is the most common extracranial solid tumor in children, accounting for 8–10% of pediatric cancers. Outcomes for patients with this tumor remain poor despite advances in multimodal therapy. Anti-GD2 monoclonal antibodies promote immune-mediated cytotoxicity by targeting the disialoganglioside GD2 on tumor cells. The addition of GM-CSF may potentiate this effect, but studies show variable efficacy and toxicity, with no consensus on optimal dosing or comparative benefit versus other cytokines.
Design/Methods:
The PRISMA guidelines were followed for this systematic review and meta-analysis and it was registered in PROSPERO (CRD420251141716). A comprehensive literature search was conducted in PubMed and Embase from inception to September 2025 to identify studies evaluating the efficacy and safety of anti-GD2 antibody therapy combined with granulocyte-macrophage colony-stimulating factor (GM-CSF) in patients with neuroblastoma. Data were synthesized using a random-effects model in R, and single-arm pooled event rates were calculated for efficacy and safety outcomes.
Results:
Thirteen studies (n = 2008) were included, evaluating hu14.18K322A (n = 172) and dinutuximab (n = 1836). The pooled objective response rate was 41% (95% CI: 0.28–0.56), with comparable efficacy for hu14.18 (43%) and dinutuximab (40%). Five studies (n = 1409) reported a pooled progression-free survival rate of 50% (95% CI: 0.21–0.79), while stable disease was observed at 37% (95% CI: 11–67%). Common adverse events included fever (31%), pain (42%), and hypotension (21%), with variable heterogeneity across studies.
Conclusions:
Anti-GD2 combined with GM-CSF shows promising efficacy and tolerable toxicity in relapsed and refractory neuroblastoma, though further studies are needed to confirm consistency across treatment settings.
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