Efficacy and Safety of Anti-GD2 Immunotherapy with Dinutuximab Beta in the Treatment of Relapsed/Refractory High-Risk Neuroblastoma

High-risk neuroblastoma (HR-NB) is associated with a poor prognosis. Standard first-line maintenance therapy with anti-disialoganglioside 2 (GD2) monoclonal antibodies, such as dinutuximab beta, has improved survival rates; however, approximately 50% of patients experience relapse and ~15% have disease that is refractory to induction therapy.

This systematic literature review aimed to evaluate response rates, survival outcomes, and safety in patients with relapsed or refractory (R/R) HR-NB receiving dinutuximab beta as maintenance therapy.

We searched the PubMed, Embase, and Cochrane Library databases and regulatory reports from inception to 1 September 2024, and included studies of patients with R/R HR-NB in which dinutuximab beta (± isotretinoin) was used as maintenance therapy and that reported objective response or survival rates. Studies of dinutuximab beta plus chemotherapy combinations were excluded.

We included nine publications/reports representing seven studies and 442 patients receiving dinutuximab beta. Across studies, the mean age was 5.1–6.4 years, and most patients were male. Reporting of response varied across studies between best response and end-of-treatment response. Best response rates with dinutuximab beta were 28.6–54.8%. All studies reported overall survival (OS), but follow-up times varied. Where reported, 3-year OS rates for patients receiving dinutuximab beta were 54–86% overall, with better OS rates reported for refractory than relapsed patients. Adverse events were frequent but manageable.

Maintenance therapy for patients with R/R HR-NB with dinutuximab beta as monotherapy or in combination with isotretinoin demonstrated efficacy and acceptable safety. Further studies are needed in patients previously treated with anti-GD2 therapies to evaluate efficacy and impact on target antigens.