Background Neuroblastoma is an extremely malignant tumor in children. For advanced or recurrent cases, existing treatment modalities are limited and efficacy remains disappointing. With the improvement in understanding of molecular biology of neuroblastoma and the development of clinical trials of targeted drug therapy, a variety of targeted therapies for neuroblastoma have appeared. Data sources All the recent literatures on targeted therapies of neuroblastoma on PubMed were searched and reviewed. Results This article reviewed targeted therapies of neuroblastoma going through clinical trials and obtained preliminary results. The features, advantages and disadvantages of targeted radiation therapy, immunotherapy, gene and pathway molecular inhibitor and angiogenesis inhibitor were discussed. Conclusion This study provides references for better understanding the current progress of targeted therapies for neuroblastoma.

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神经母细胞瘤靶向治疗的应用及前景

背景 神经母细胞瘤是儿童中一种极其恶性的肿瘤。对于晚期或复发病例，现有的治疗方式有限，疗效仍然令人失望。随着对神经母细胞瘤分子生物学认识的提高和靶向药物治疗临床试验的开展，出现了多种针对神经母细胞瘤的靶向治疗药物。数据来源检索并查阅了PubMed上所有关于神经母细胞瘤靶向治疗的最新文献。结果 本文综述了正在进行临床试验的神经母细胞瘤靶向治疗，并取得了初步结果。讨论了靶向放射治疗、免疫治疗、基因与通路分子抑制剂和血管生成抑制剂的特点、优缺点。