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Abstract Details

Efficacy and Safety of Anti-GD2 Monoclonal Antibodies Combined with GM-CSF in Relapsed and Refractory Neuroblastoma: A Systematic Review and Meta-analysis
Child Neurology and Developmental Neurology
P9 - Poster Session 9 (5:00 PM-6:00 PM)
8-008
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.
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.
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.
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.

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.


Authors/Disclosures
Muaz Ahmed, MBBS
PRESENTER
Mr. Ahmed has nothing to disclose.
Anas Nasir, MBBS Mr. Nasir has nothing to disclose.
Dania Hussain Miss Hussain has nothing to disclose.
Suleman Saeed, MBBS Mr. Saeed has nothing to disclose.
Hamid Bin Tariq, MBBS Mr. Bin Tariq has nothing to disclose.
Izma Kashif, MBBS Miss Kashif has nothing to disclose.
Zoha Kashif, MBBS Ms. Kashif has nothing to disclose.
Tooba Ali, MBBS Dr. Ali has nothing to disclose.
Maham Amer, MBBS Dr. Amer has nothing to disclose.
Muhammad Ali E. Bhatti, MBBS Mr. Bhatti has nothing to disclose.
Abdul Rehman, MBBS Mr. Rehman has nothing to disclose.
Zainab Tanveer, MBBS Dr. Tanveer has nothing to disclose.
Jaweria Sharif, MBBS Miss Sharif has nothing to disclose.
Awais Akhtar, MBBS No disclosure on file
Muhammad Owais Mazhar Dr. Mazhar has nothing to disclose.
Muhammad Ali Bin Jabir, MBBS Dr. Jabir has nothing to disclose.