Anti-GD2 immunotherapy for newly-diagnosed or recurrent high-risk neuroblastoma

Abstract

Remarkable progress has been made in the treatment of high-risk neuroblastoma with anti-GD2 immunotherapy. However, its efficacy and safety, especially in relapsed cases, remain unclear. We retrospectively analyzed nine patients who received anti-GD2 immunotherapy at our hospital between October 2021 and October 2024. Three patients received anti-GD2 antibody as maintenance therapy after first-line treatment (maintenance group), while six received salvage therapy for relapse (relapse group). The median age at therapy initiation was two years in the maintenance group and three years in the relapse group. The median number of treatment cycles was 6 and 3.5, respectively. Notably, four patients in the relapse group discontinued treatment due to progressive disease (PD). Hematologic toxicities included grade ≥3 thrombocytopenia, which tended to be more frequent in the relapse group than in the maintenance group. With respect to non-hematologic toxicities, neither group experienced grade ≥3 infusion reactions or capillary leak syndromes. The best overall responses were complete response (CR) in two patients and partial response (PR) in one patient in the maintenance group, compared to stable disease (SD) in one patient and PD in five patients in the relapse group. Among the three patients who underwent cord blood transplantation, one patient in the maintenance group completed all cycles without serious adverse events, while two patients in the relapse group discontinued treatment due to PD. Therefore, anti-GD2 immunotherapy is applicable to recurrent cases with acceptable safety, although its efficacy is limited. Further studies are warranted to determine the optimal timing and combination strategies for anti-GD2 immunotherapy for high-risk neuroblastoma.