Dana-Faber Cancer Institute, Department of Pediatric Oncology, Boston, MA, USA.
Expert Opin Pharmacother. 2010 Jun;11(9):1467-78. doi: 10.1517/14656566.2010.482100.
Neuroblastoma, a tumor of the sympathetic nervous system, is the most common extracranial solid tumor of early life. High risk disease in older children remains a therapeutic challenge, despite high-intensity therapy with correspondingly significant short- and long-term toxicities.
We have reviewed therapy for neuroblastoma over the last three decades. This includes cytotoxic chemotherapy, immunotherapy, radionuclides, antiangiogenic compounds, and molecularly targeted agents. We provide a perspective on the incorporation of these drugs into therapy for neuroblastoma.
The reader will gain a better understanding of these novel agents and their targets in neuroblastoma. The reader will also gain insight into the need to define through sequential, carefully designed clinical trials, the roles and toxicities of these therapies, especially if the combination of targeted and conventional cytotoxic agents is used.
Advanced-stage neuroblastoma in older infants and children remains a disease that is difficult to cure. New, targeted agents may improve both the therapeutic index and the outcome, but are, for the most part, in early development and present a challenge for clinical trial design given both the rarity of this disease and its responsiveness (albeit incomplete) to currently used cytotoxic agents.
神经母细胞瘤，一种交感神经系统的肿瘤，是婴儿期最常见的颅外实体瘤。尽管采用高强度治疗，伴有相应的显著短期和长期毒性，但大龄儿童的高危疾病仍然是一个治疗挑战。
我们回顾了过去三十年的神经母细胞瘤治疗方法。这包括细胞毒性化疗、免疫疗法、放射性核素、抗血管生成化合物和分子靶向药物。我们提供了将这些药物纳入神经母细胞瘤治疗的观点。
读者将更好地了解这些新型药物及其在神经母细胞瘤中的靶点。读者还将深入了解需要通过连续、精心设计的临床试验来确定这些疗法的作用和毒性的必要性，特别是如果靶向和常规细胞毒性药物联合使用。
大龄婴儿和儿童的晚期神经母细胞瘤仍然是一种难以治愈的疾病。新的靶向药物可能会改善治疗指数和结果，但它们在很大程度上仍处于早期开发阶段，并且由于这种疾病的罕见性及其对目前使用的细胞毒性药物的反应性（尽管不完整），对临床试验设计提出了挑战。
Zotero 插件