Biologically targeted therapeutics in pediatric brain tumors.
Biologically targeted therapeutics in pediatric brain tumors.
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DOI：
10.1016/j.pediatrneurol.2012.02.005
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发表时间：
2012-04
影响因子：
3.8
通讯作者：
Packer RJ
中科院分区：
文献类型：
--
作者：
Nageswara Rao AA;Scafidi J;Wells EM;Packer RJ
儿童脑肿瘤通常难以治愈，并且在采用传统治疗方式（包括神经外科手术、常规化疗和放疗）治疗时，发病率很高。在过去的二十年中，人们对癌症发病机制中的肿瘤发生、分子生长途径和免疫机制有了更清晰的了解，为治疗开辟了有希望的途径。使用新型生物制剂治疗各种儿童脑肿瘤的儿科临床试验正在进行中，包括高级别和低级别胶质瘤和胚胎肿瘤。随着这些药物的治疗潜力得到评估，它们的毒性特征也得到了更好的了解。与传统化疗相比，这些药物可能具有更好的中枢神经系统渗透性和更低的毒性。对于婴儿和年幼的儿童，与传统化疗和放疗相比，生物制剂可能具有相同或更好的功效，并且可以减少传统疗法对发育中的大脑的有害副作用。回顾了与儿科脑肿瘤有关的分子途径、针对这些途径的药物以及当前的临床试验。随后将讨论相关的神经毒性。需要做大量的工作来确定这些药物单独和联合使用的功效，但儿科神经科医生应该了解这些药物及其基本原理。
Pediatric brain tumors are often difficult to cure and involve significant morbidity when treated with traditional treatment modalities, including neurosurgery, conventional chemotherapy, and radiotherapy. During the past two decades, a clearer understanding of tumorigenesis, molecular growth pathways, and immune mechanisms in the pathogenesis of cancer has opened up promising avenues for therapy. Pediatric clinical trials with novel biologic agents are underway to treat various pediatric brain tumors, including high and low grade gliomas and embryonal tumors. As the therapeutic potential of these agents undergoes evaluation, their toxicity profiles are also becoming better understood. These agents have potentially better central nervous system penetration and lower toxicity profiles compared with conventional chemotherapy. In infants and younger children, biologic agents may prove to be of equal or greater efficacy compared with traditional chemotherapy and radiation therapy, and may reduce the deleterious side effects of traditional therapeutics on the developing brain. Molecular pathways implicated in pediatric brain tumors, agents that target these pathways, and current clinical trials are reviewed. Associated neurologic toxicities will be discussed subsequently. Considerable work is needed to establish the efficacy of these agents alone and in combination, but pediatric neurologists should be aware of these agents and their rationale.
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影响因子：
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作者：
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通讯作者：
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