Jenabzade Alireza, Alavi Samin, Aminasnafi Ali
Hematology and Oncology Research Center, Non-communicable Diseases Research Institute, Shahid Sadoughi University of Medical Sciences, Yazd, Iran.
Department of pediatric hematology oncology, Research Development Center, Shahid Sadoughi Hospital, School of Medicine, Shahid Sadoughi University of Medical Sciences, Yazd, Iran.
Iran J Child Neurol. 2025 Jun 25;19(3):9-29. doi: 10.22037/ijcn.v19i3.48040. eCollection 2025 Summer.
Neuroblastoma is one of the most common pediatric cancers, predominantly affecting young children. Despite progress in initial treatments, high-risk cases remain challenging due to frequent relapse or resistance, with long-term survival for relapsed or refractory neuroblastoma below 20%. This highlights an urgent need for novel therapies. Emerging approaches such as GD2-targeted immunotherapy with monoclonal antibodies like dinutuximab, CAR-T cell therapy, 131I-MIBG and Lutetium-177-Dotatate radionuclide treatments, metronomic chemotherapy, oncolytic virotherapy, and tailored chemotherapy are showing promise, with autologous stem cell transplantation (ASCT) becoming integral to multimodal regimens. However, challenges persist, including treatment-related toxicity, tumor resistance, and the logistical limitations of personalized medicine. The future of neuroblastoma treatment lies in exploiting genomic profiling, biomarkers, and combinatorial strategies like immunotherapy paired with radionuclide therapy. Rigorous clinical trials will be key to refining these innovations and establishing protocols for widespread use. In summary, advancements in therapy offer hope, yet achieving durable remissions and improved survival still demands intensive research innovation to address current gaps and resistance mechanisms in this complex pediatric malignancy.
神经母细胞瘤是最常见的儿童癌症之一,主要影响幼儿。尽管初始治疗取得了进展,但高危病例由于频繁复发或耐药性,仍然具有挑战性,复发或难治性神经母细胞瘤的长期生存率低于20%。这凸显了对新型疗法的迫切需求。诸如使用地努图希单抗等单克隆抗体进行的GD2靶向免疫疗法、嵌合抗原受体T细胞(CAR-T)疗法、131I-间碘苄胍(MIBG)和镥-177-奥曲肽放射性核素治疗、节拍化疗、溶瘤病毒疗法以及个性化化疗等新兴方法显示出前景,自体干细胞移植(ASCT)已成为多模式治疗方案不可或缺的一部分。然而,挑战依然存在,包括治疗相关毒性、肿瘤耐药性以及个性化医疗的后勤限制。神经母细胞瘤治疗的未来在于利用基因组分析、生物标志物以及免疫疗法与放射性核素疗法等联合策略。严格的临床试验将是完善这些创新并建立广泛应用方案的关键。总之,治疗方面的进展带来了希望,但要实现持久缓解和提高生存率,仍需要深入的研究创新来解决这种复杂儿童恶性肿瘤当前存在的差距和耐药机制。