Goldsby Robert E, Matthay Katherine K
Division of Pediatric Hematology/Oncology, University of California, San Francisco 94143-0106, USA.
Paediatr Drugs. 2004;6(2):107-22. doi: 10.2165/00148581-200406020-00004.
Neuroblastoma is the most common extra-cranial solid tumor in children and has a heterogeneous clinical presentation and course. Clinical and biologic features of this disease have been used to develop risk-based therapy. Patients with low-risk disease can be treated with surgery alone. Patients with intermediate-risk features have an excellent prognosis after treatment with surgery and a relatively short course of standard dose chemotherapy. Unfortunately, most children with neuroblastoma present with advanced disease. More than 60% of patients with high-risk features will succumb to their disease despite intensive therapy including a myeloablative consolidation. Research efforts to understand the biologic basis of neuroblastoma and to identify new, more effective therapies are essential to improve the outcome for these children.
神经母细胞瘤是儿童最常见的颅外实体瘤，临床表现和病程具有异质性。该疾病的临床和生物学特征已被用于制定基于风险的治疗方案。低风险疾病患者可仅接受手术治疗。具有中风险特征的患者在接受手术和相对较短疗程的标准剂量化疗后预后良好。不幸的是，大多数神经母细胞瘤患儿就诊时已处于疾病晚期。超过60%具有高风险特征的患者尽管接受了包括清髓性巩固治疗在内的强化治疗，仍会死于该疾病。了解神经母细胞瘤生物学基础并确定新的、更有效的治疗方法的研究工作对于改善这些儿童的治疗结局至关重要。
