Improving the outcome for children with cancer: Development of targeted new agents.
Improving the outcome for children with cancer: Development of targeted new agents.
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DOI：
10.3322/caac.21273
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发表时间：
2015-05
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影响因子：
--
通讯作者：
Adamson PC
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作者：
Adamson PC
在过去的60年里，癌症儿童的预后得到了显著改善，今天超过80%的患者成为5年生存者。尽管取得了这一进展，但癌症仍然是美国儿童疾病死亡的主要原因，并且显著的短期和长期治疗毒性继续影响大多数癌症儿童。靶向新药的开发为儿童提供了潜在的更有效和毒性更小的治疗的前景。自从伊马替尼甲磺酸盐被引入费城染色体阳性急性淋巴细胞白血病儿童的治疗，改变了其结果，十多年来，一系列靶向药物已经在儿科癌症患者中进行了研究。从这些研究中获得的早期经验教训包括更好地了解这些药物在儿童中的不良事件特征，开发儿科特定制剂的挑战，以及继续依赖于成人癌症适应症的成功开发。美国癌症儿童的合作研究基础设施处于有利地位，可以将新的治疗方法推进到一系列罕见和超罕见儿童癌症的临床研究中。在目标发现和验证方面投入更多的资源，可以帮助推动急需的新的、更有效的癌症儿童治疗方法的开发。
The outcome for children with cancer has improved significantly over the past 60 years, with greater than 80% of patients today becoming 5-year survivors. Despite this progress, cancer remains the leading cause of death from disease in children in the United States, and significant short-term and long-term treatment toxicities continue to impact the majority of children with cancer. The development of targeted new agents offers the prospect of potentially more effective and less toxic treatment for children. More than a decade since imatinib mesylate was introduced into the treatment of children with Philadelphia chromosomepositive acute lymphoblastic leukemia, transforming its outcome, a range of targeted agents has undergone study in pediatric cancer patients. Early lessons learned from these studies include a better understanding of the adverse event profile of these drugs in children, the challenge of developing pediatric-specific formulations, and the continued reliance on successful development for adult cancer indications on pediatric drug development. The collaborative research infrastructure for children with cancer in the United States is well positioned to advance novel treatments into clinical investigations for a spectrum of rare and ultra-rare childhood cancers. A greater investment of resources in target discovery and validation can help drive much needed development of new, more effective treatments for children with cancer.