Division of Pediatric Hematology/Oncology, Department of Oncological Sciences, University of Utah, 2000 Circle of Hope, Salt Lake City, UT 84112, USA.
Expert Opin Pharmacother. 2010 Jul;11(10):1621-32. doi: 10.1517/14656566.2010.484019.
Pediatric acute lymphoblastic leukemia (ALL) represents one of the best examples of progress in disease treatment and improved outcome based in part upon the incorporation of the principles of pharmacogenomics. Throughout the past several decades, clinical scientists have continued to refine risk stratification in clinical trials with the understanding that individual patients have different subtypes of pediatric ALL that will respond to therapy in different, but predictable ways.
Discussed in this review are the most significant findings from pharmacogenomic studies of pediatric ALL from 1989 to the present. Pharmacogenomic studies related to the drugs commonly used to treat pediatric ALL are covered in detail, including an emphasis on both genome-wide and candidate gene/pathway approaches.
Readers of this paper will acquire a detailed understanding of how pharmacogenomic studies can be integrated into clinical trials, in addition to some of the discrepancies still present in the field.
The outcome for children with pediatric ALL has improved greatly, and this is in part due to the successful integration of data from pharmacogenomic studies into clinical trials.
儿科急性淋巴细胞白血病(ALL)代表了疾病治疗进展和改善结果的最佳范例之一,部分原因是基于药物基因组学原理的应用。在过去的几十年中,临床科学家一直在临床试验中不断完善风险分层,他们的理解是,每个患者的儿科 ALL 都有不同的亚型,对治疗的反应也不同,但具有可预测性。
本文讨论了从 1989 年至今儿科 ALL 药物基因组学研究中最重要的发现。详细介绍了与儿科 ALL 常用治疗药物相关的药物基因组学研究,重点介绍了全基因组和候选基因/途径方法。
阅读本文的读者将详细了解如何将药物基因组学研究整合到临床试验中,以及该领域目前仍存在的一些差异。
儿科 ALL 患儿的预后有了很大的改善,这在一定程度上是由于成功地将药物基因组学研究的数据整合到临床试验中。