Department of Oncology, University College London Hospitals NHS Foundation Trust, 250 Euston Road, London NW1 2PG, UK.
Future Oncol. 2012 Jul;8(7):839-58. doi: 10.2217/fon.12.70.
Neuroblastoma is a complex disease with many contradictions and challenges. It is, by and large, a cancer of babies and preschool children, but it does occur, albeit increasingly rarely, in older children, adolescents and young adults. The prognosis is very variable, with outcome related to age, stage and molecular pathology. Neuroblastoma may behave in an almost benign way, with spontaneous regression in some infants, but the majority of older patients have high-risk disease, which is usually fatal, despite best current treatments. As a rare disease, international collaboration is essential to run clinical trials of adequate statistical power to answer important questions in a reasonable time frame. High-risk disease requires multimodality therapy including chemotherapy, surgery and radiotherapy as well as biological and immunological treatments for optimal outcomes. Innovative treatment approaches, sometimes associated with appreciable toxicity, offer hope for the future but, despite parental wishes, cannot be generally implemented without adequate assessment in clinical trials.
神经母细胞瘤是一种复杂的疾病，存在诸多矛盾和挑战。它主要发生在婴儿和学龄前儿童，但也会在年龄较大的儿童、青少年和年轻人中发生，尽管越来越罕见。预后差异很大，与年龄、分期和分子病理学有关。神经母细胞瘤的表现可能近乎良性，一些婴儿会自发消退，但大多数年龄较大的患者患有高危疾病，尽管采用了目前最好的治疗方法，但通常仍会致命。作为一种罕见疾病，开展具有足够统计效力的临床试验以在合理的时间框架内回答重要问题，国际合作至关重要。高危疾病需要采用多种方法治疗，包括化疗、手术和放疗，以及生物和免疫治疗，以获得最佳结果。创新的治疗方法有时会伴随明显的毒性，为未来带来希望，但尽管家长有此愿望，如果未经临床试验充分评估，也不能普遍实施。
