Anti-GD2 Based Immunotherapy Prevents Late Events in High-Risk Neuroblastoma Patients over 18 Months at Diagnosis

• Published in: Cancers Vol. 13; no. 19; p. 4941
• Main Authors: Tas, Michelle L., Dootjes, Lisa W., Fiocco, Marta, de Krijger, Ronald R., Dierselhuis, Miranda P., van Eijkelenburg, Natasha K. A., van Grotel, Martine, Kraal, Kathelijne C. J. M., Peek, Annemarie M. L., Tytgat, Godelieve A. M., van Noesel, Max M.
• Format: Journal Article
• Language: English
• Published: Basel MDPI AG 30.09.2021; MDPI
• Subjects: anti-GD2; Cerebrospinal fluid; Chemotherapy; Diagnosis; Granulocyte-macrophage colony-stimulating factor; high-risk; Immunotherapy; Interleukin 2; metastatic; Monoclonal antibodies; Neuroblastoma; Patients; Pediatrics; Stem cells; Survival
• ISSN: 2072-6694; 2072-6694
• DOI: 10.3390/cancers13194941

Background: Anti-GD2 based immunotherapy has improved overall (OS) and event free survival (EFS) for high-risk neuroblastoma (HR-NBL) patients. Here, we evaluate the long-term efficacy of anti-GD2 immunotherapy in combination with isotretinoin, GM-CSF, and IL-2. Methods: Dutch HR-NBL patients treated with immunotherapy according to the COG-ANBL0032 protocol (n = 47) were included and compared to historical controls (n = 37) treated with single-agent isotretinoin maintenance therapy. Survival time was calculated from start of the maintenance therapy. Results: The study and control group were similar concerning baseline characteristics. In the complete cohort, 5 year OS was 64 ± 7% and 49 ± 8% for the immunotherapy group and the control group, respectively (p = 0.16). Five year EFS was 57 ± 7% and 41 ± 8%, respectively (p = 0.16). In the subgroup of patients ≥ 18 months, 5-yr OS was 63 ± 8% and 39 ± 9, respectively (p = 0.04) and EFS 54 ± 8% and 29 ± 8%, respectively (p = 0.05). Landmark analysis for EFS with landmark point at 6 months after start of maintenance suggests a larger effect on the prevention of late than early events. Conclusions: This study is the first to confirm the results of the COG-ANBL0032 study in a cohort treated with a different induction regimen. Anti-GD2 immunotherapy prevents late events, most significantly in patients older than 18 months of age at diagnosis.