国外专家答疑｜神经母细胞瘤

        葵花子从读者来信中精选了5个关于神经母细胞瘤的问题向国外专家咨询，以下是这些问题和专家回复：

1

        问： 术后有残留是否可以带瘤生存？（例如：如椎管椎管有残留没有手术，带瘤生存的希望大不大？）   

        回复：可以。尤其是对于那种局限性的肿瘤并且具有良好的生物学特征的患者。

2

        问：目前质子放疗和其他放疗那个更适合神母患者，两者的优势和缺点各是什么？

        回复：目前没有证据显示质子放疗比其他传统的放疗更适合神母患者。

3

        问：国际上四期有没有异体移植治愈的案例，自体移植和异体移植的区别和优势？

        回复：所有的大量的儿童肿瘤三期的试验均证明了自体移植对于高危的神母患者的有效性。从经历了2-3个周期诱导化疗后的病人中提取的干细胞,将它们再输注于骨髓自体移植可救治致命剂量的化疗带来的影响。

        异体移植（应用的供体非患者自身）目前多用于肿瘤复发的病人，但是使用这种方法的经验非常有限，而且可以预期到更多的副作用发生。

4

        问：神母4期的孩子复发后有什么最新的治疗手段，效果如何？复发后的治愈率（5年生存率）是多少？

        回复：对于经历复发的高危神经母细胞瘤患儿目前没有一个标准的治疗方案。治疗需要个体化，并且取决于复发和疾病负担的模式。

        治疗方案包括碘131-间位碘卞胍（131I-MIBG）显像疗法,靶向的试验性治疗,常规的利用化疗/放疗和免疫的治疗疗法。进行复发肿瘤的分子表达谱的分析来检查是否有肿瘤基因通道的突变来看是否有想匹配的药物是很有意义的（例如克里唑蒂尼对于间变性淋巴瘤激酶筛选突变的作用）。

5

        问：孩子出现肠漏，一直不能吃东西，不能化疗，治肠漏，可能要几个月才能好，能否有其他方式控制肿瘤病情发展？

        回复：即使孩子出现肠漏，对于高危的神母患者化疗仍不应拖延。应竭尽一切去坚持化疗，使用静脉输入营养液(静脉营养)直到进行肠道治疗。

        英语原文：

        Question: If there is a postoperative residual tumor, could the patient live with the tumor?( For example, if there is a residual tumor in the spinal canal that not be operated, is the hope big for living with the tumor?)

        Answer: Yes. Especially if the tumor is localized and has favorable biological features.

        Question: Between the proton radiotherapy and other kind of radiotherapiy, which one is more suitable for neuroblastoma patients at present? What are their respective advantages and disadvantages?

        Answer: There is no proven advantage at this time to proton beam radiotherapy versus more conventional IMRT.

        Question: In the international is there any stage IV case that is cured by allotransplantation? What’s the difference between autotransplantation and allotransplantation and what’re their respective advantages?

        Answer: All of the large Phase 3 Children’s Oncology Group trials have tested the efficacy or autotransplantion for children with high-risk neuroblastoma in the upfront setting. The stem cells are harvested form the patient after 2-3 cycles of induction chemotherapy and then re-infused at time of myeloablative autotransplantation to rescue the body from the effects of the lethal doses of chemotherapy.

        Allo-transplantation (using a donor that is not “self”) has been tested mostly in the setting of relapse- there is very limited experience using this approach and many more side effects can be expected.

        Question: What is the latest treatment for the children whose neuroblastoma stage IV have already showed recurrence? How is the effect of the treatment? How much is the cure rate (5-year survival rate) after recurrence?

         Answer: There is no standard treatment for children with high-risk neuroblastoma who suffer a relapse. This needs to be individualized and often depends on the pattern of relapsed and disease burden. Approaches include I131MIBG therapy, targeted investigational therapies, and conventional approaches that utilized chemo/radiation and immunotherapy. There is a significant effort to perform molecular profiling on the tumor at relapse to see if there are targetable mutations in cancer genes for which there may be matched drugs that can be used (an example is crizotinib when there is an ALK mutation).

        Question: If the children appear leaky gut and always can't eat or chemotherapy, it takes to several months to cure leaky gut, so whether there are other ways to control tumor progression?

        Answer: Even in the setting of a “leaky gut,” chemotherapy should not be delayed in the setting of high-risk neuroblastoma. Every effort should be made to continue chemotherapy and to sue hyperalimentation (IV nutrition) until the gut heals.

        翻译：再见图安
        向日葵编缉：小东，菠萝