Objective  To systematically review the safety of disialoganglioside (GD2) targeted therapies in the treatment of pediatric neuroblastoma (NB).

Methods  PubMed, Embase, Cochrane Library, CNKI, WanFang Data, SinoMed databases and ClinicalTrials.gov clinical trial registration system were electronically searched to collect clinical studies of GD2-targeted therapies for pediatric NB from inception to November 30, 2024. Two reviewers independently screened the literature, extracted data and assessed the risk of bias of the included studies. Meta-analysis was performed by using R 4.3.1 software.

Results  A total of 21 studies involving 2,438 patients were included. Meta-analysis results showed that the overall incidence (95%CI) of serious adverse drug events (SADEs) in GD2-targeted therapies for pediatric NB was 51.67% (35.43% to 67.92%). The serious fever and alanine aminotransferase (ALT) increase were common with the incidence (95%CI) of 7.80% (0.80% to 18.85%) and 5.10% (2.02% to 10.55%) respectively. The incidences (95%CI) of grade 3 to 5 adverse drug reactions (ADRs): fever 18.81% (8.00% to 29.61%), pain 30.32% (16.21% to 44.44%), hypokalemia 18.96% (6.44% to 31.49%), ALT increased 10.41% (2.96% to 17.86%), hypotension 8.48% (3.78% to 13.18%), anemia 16.62% (5.97% to 27.26%), platelet count decreased 16.46% (5.55% to 27.37%), lymphocyte count decreased 10.42% (0.97% to 26.09%), neutrophil count decreased 13.37% (3.12% to 23.62%). Among the intervention subgroups, the incidences of grade 3 to 5 ADRs were relatively low in dinutuximab β group and GD2-chimeric antigen receptor T cells (GD2 CART) group.

Conclusions  The incidences of SADEs and grade 3 to 5 ADRs are high in pediatric NB patients receiving GD2-targeted therapy, which should be paid attention to. The safety profile of GD2 targeted therapies under development for pediatric NB are even better than those of GD2 targeted drugs already on the market, especially GD2 CART, which shows good safety potential.

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