AUTHOR=Di Ruscio Valentina , Carboni Alessia , Del Baldo Giada , De Pasquale Maria Debora , Valente Paola , Di Giannatale Angela , Serra Annalisa , De Vito Rita , Mastronuzzi Angela , Quintarelli Concetta , De Angelis Biagio , Milano Giuseppe Maria , Russo Ida 

TITLE=Prolonged overall survival in a child with multimetastatic retinoblastoma treated with anti-GD2 monoclonal antibody dinutuximab beta: a case report

JOURNAL=Frontiers in Oncology

VOLUME=Volume 15 - 2025

YEAR=2025

URL=https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1665968

DOI=10.3389/fonc.2025.1665968

ISSN=2234-943X

ABSTRACT=High-dose chemotherapy with autologous stem cell rescue has improved outcomes in patients with metastatic retinoblastoma (RB). However, significant short- and long-term toxicities—especially in very young children with a constitutional RB1 gene mutation—highlight the need for alternative therapeutic strategies. Monoclonal antibodies targeting tumor-associated antigens such as GD2 have emerged as promising agents in this setting. We report the case of a 2-year-old child diagnosed with extensive left-eye retinoblastoma and massive extraocular dissemination at presentation. The patient was treated with systemic conventional and high-dose chemotherapy combined with intrathecal Topotecan. As consolidation therapy, the child received three courses of the anti-GD2 monoclonal antibody Dinutuximab beta over a 10-day schedule. The patient achieved complete remission and remains disease-free six years after the initial diagnosis. This case suggests that anti-GD2 immunotherapy, used as consolidation treatment, may improve the prognosis of patients with advanced retinoblastoma and potentially reduce the toxicity associated with standard therapies. Further clinical investigation is warranted to validate these findings.