Article: 4SCAR-GD2-modified T-cell therapy in neuroblastoma with amplification: A case report with over 4-year follow-up data

Introduction: Neuroblastoma (NB) is the most common extracranial solid tumor among children. The 5-year event-free survival rate for high-risk (HR) NB is still poor, especially for patients with advanced NB with gene amplification. Chimeric antigen receptor T (CAR-T) cell therapy is a new treatment for HR-NB.

Case Presentation: A 55-month-old boy with stage IV HR-NB received 4th-generation CAR-T cells that target disialoganglioside GD2, as consolidation maintenance treatment after intensive chemotherapy, surgery, and autologous stem-cell transplantation. As of February 2019, his CAR-T follow-up time was 37.5 months, indicating prolonged survival. Cranial MRI and ultrasound showed no mass; I-metaiodobenzylguanidine (I-MIBG) scan was negative.

Conclusion: GD2-CAR-T cells may be an effective treatment option for NB patients with amplification.

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4SCAR-GD2-modified T-cell Therapy in Neuroblastoma with Amplification: A Case Report with over 4-year Follow-up Data