Center for Neural Development and Disease, and Department of Pediatrics, University of Rochester School of Medicine and Dentistry, 601 Elmwood Avenue, Rochester, NY 14642, USA.
Paediatr Drugs. 2011 Aug 1;13(4):245-55. doi: 10.2165/11591630-000000000-00000.
Neuroblastoma is the most common extracranial solid tumor of childhood. It accounts for 15% of pediatric cancer deaths. Children with high-risk disease have a 3-year event-free survival rate of only 20%. Chemotherapy is the mainstay of treatment in children with advanced neuroblastoma. The aim of this article was to review and critically evaluate the pharmacotherapy of neuroblastoma, using peer reviewed and review literature from 2000-11. All peer reviewed, published human subject studies of therapy for neuroblastoma in children were included. Animal model and in vitro studies were included only if they added to the understanding of the mechanism of a proposed or existing human neuroblastoma therapy. Current therapeutic options for neuroblastoma involve insufficient differentiation of normal from neoplastic tissue. Critically needed new approaches will increasingly exploit targeting of therapy for unique characteristics of the neuroblastoma cell. Pharmacotherapy for neuroblastoma still suffers from an inadequate therapeutic window. Enhancement of toxicity for tumor and safety for normal tissues will entail innovation in targeting neuroblastoma cells and rescuing or protecting normal tissue elements.
神经母细胞瘤是儿童期最常见的颅外实体瘤。它占儿童癌症死亡人数的 15%。患有高危疾病的儿童，3 年无事件生存率仅为 20%。化疗是治疗晚期神经母细胞瘤儿童的主要方法。本文旨在回顾和批判性评估神经母细胞瘤的药物治疗，使用了 2000-11 年的同行评议和综述文献。所有经过同行评议、发表的关于儿童神经母细胞瘤治疗的人类研究都包括在内。只有当动物模型和体外研究有助于理解拟议或现有的人类神经母细胞瘤治疗的机制时，才包括这些研究。目前神经母细胞瘤的治疗选择涉及正常组织与肿瘤组织之间的分化不足。迫切需要新的方法，将越来越多地利用针对神经母细胞瘤细胞独特特征的治疗靶向。神经母细胞瘤的药物治疗仍然存在治疗窗口不足的问题。提高肿瘤的毒性和正常组织的安全性需要在针对神经母细胞瘤细胞和挽救或保护正常组织成分方面进行创新。
