Maris John M, Hogarty Michael D, Bagatell Rochelle, Cohn Susan L
Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA 19104-4318, USA.
Lancet. 2007 Jun 23;369(9579):2106-20. doi: 10.1016/S0140-6736(07)60983-0.
The clinical hallmark of neuroblastoma is heterogeneity, with the likelihood of cure varying widely according to age at diagnosis, extent of disease, and tumour biology. A subset of tumours will undergo spontaneous regression while others show relentless progression. Around half of all cases are currently classified as high-risk for disease relapse, with overall survival rates less than 40% despite intensive multimodal therapy. This Seminar focuses on recent advances in our understanding of the biology of this complex paediatric solid tumour. We outline plans for the development of a uniform International Neuroblastoma Risk Group (INRG) classification system, and summarise strategies for risk-based therapies. We also update readers on new discoveries related to the underlying molecular pathogenesis of this tumour, with special emphasis on advances that are translatable to the clinic. Finally, we discuss new approaches to treatment, including recently discovered molecular targets that might provide more effective treatment strategies with the potential for less toxicity.
神经母细胞瘤的临床特征是异质性，根据诊断时的年龄、疾病范围和肿瘤生物学特性，治愈的可能性差异很大。一部分肿瘤会自发消退，而其他肿瘤则会持续进展。目前，约一半的病例被归类为疾病复发高危，尽管进行了强化多模式治疗，总体生存率仍低于40%。本研讨会聚焦于我们对这种复杂儿科实体瘤生物学理解的最新进展。我们概述了统一国际神经母细胞瘤风险组（INRG）分类系统的制定计划，并总结了基于风险的治疗策略。我们还向读者介绍了与该肿瘤潜在分子发病机制相关的新发现，特别强调了可转化至临床的进展。最后，我们讨论了新的治疗方法，包括最近发现的可能提供更有效治疗策略且毒性可能更低的分子靶点。
