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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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7331334 | PMC |
| http://dx.doi.org/10.1002/ped4.12181 | DOI Listing |
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