Davidoff Andrew M
Department of Surgery, St Jude Children's Research Hospital, Memphis, Tennessee, USA.
Semin Pediatr Surg. 2012 Feb;21(1):2-14. doi: 10.1053/j.sempedsurg.2011.10.009.
Neuroblastoma is a heterogeneous disease; tumors can spontaneously regress or mature, or display an aggressive, therapy-resistant phenotype. Increasing evidence indicates that the biological and molecular features of neuroblastoma significantly influence and are highly predictive of clinical behavior. Because of this, neuroblastoma has served as a paradigm for biological risk assessment and treatment assignment. Most current clinical studies of neuroblastoma base therapy and its intensity on a risk stratification that takes into account both clinical and biological variables predictive of relapse. For example, surgery alone offers definitive therapy with excellent outcome for patients with low-risk disease, whereas patients at high risk for disease relapse are treated with intensive multimodality therapy. In this review recent advances in the understanding of the molecular genetic events involved in neuroblastoma pathogenesis are discussed, and how they are impacting the current risk stratification and providing potential targets for new therapeutic approaches for children with neuroblastoma. In addition, the results of significant recent clinical trials for the treatment of neuroblastoma are reviewed.
神经母细胞瘤是一种异质性疾病；肿瘤可自发消退或成熟，或表现出侵袭性、抗治疗表型。越来越多的证据表明，神经母细胞瘤的生物学和分子特征对临床行为有显著影响，且具有高度预测性。因此，神经母细胞瘤已成为生物学风险评估和治疗分配的范例。目前大多数神经母细胞瘤临床研究的治疗方法及其强度基于一种风险分层，该分层考虑了预测复发的临床和生物学变量。例如，对于低风险疾病患者，单纯手术即可提供具有良好预后的确定性治疗，而疾病复发风险高的患者则接受强化多模式治疗。在这篇综述中，将讨论在神经母细胞瘤发病机制中涉及的分子遗传事件的最新研究进展，以及它们如何影响当前的风险分层，并为神经母细胞瘤患儿的新治疗方法提供潜在靶点。此外，还将综述近期治疗神经母细胞瘤的重要临床试验结果。
