Purpose: To develop a Latin American expert consensus to support, guide, and accelerate the approval and equitable access to anti-GD2 immunotherapy for patients with high-risk neuroblastoma (HR-NB) in the region. Survival rates for HR-NB have improved with the incorporation of anti-GD2 immunotherapy; however, despite the approval of two commercial anti-GD2 therapies in two countries, access remains limited in Latin America.
Methods and results: A systematic review was conducted of studies published from January 2004 through February 2025. Of 401 studies identified, 29 met inclusion criteria and served as the basis for four expert consensus statements on the use of anti-GD2 immunotherapy: (1) Newly diagnosed HR-NB maintenance therapy: Anti-GD2 immunotherapy as maintenance treatment for all newly diagnosed HR-NB patients is strongly recommended to maximize event-free survival and reduce the need for morbid and costly salvage therapies. (2) Relapsed/refractory NB: Early integration (at first relapse) of anti-GD2 immunotherapy in salvage treatment is recommended. (3) Role of high-dose chemotherapy with autologous stem-cell transplant (ASCT): Some argue that anti-GD2 immunotherapy obviates the need for ASCT. However, until results of randomized trials omitting ASCT are available, the panel recommends continued use of ASCT where it is available. (4) Newly diagnosed HR-NB: incorporation of anti-GD2 during induction/consolidation: Pilot studies using early anti-GD2 immunotherapy plus chemotherapy showed promising results but is not recommended until confirmed by larger studies with well-defined control arms.
Conclusion: This consensus highlights the urgent need to expand access to anti-GD2 therapy in Latin America as maintenance therapy for newly diagnosed patients with HR-NB and patients with relapsed or refractory neuroblastoma. Collaboration between health policymakers, the pharmaceutical industry, the Global Platform for Access to Childhood Cancer Medicines, and the pediatric oncology community is essential to address this challenge.