Suarez-Kurtz Guilherme
Instituto Nacional de Cancer, Rio de Janeiro, RJ, BR.
Rede Nacional de Farmacogenetica, Rio de Janeiro, RJ, BR.
Clinics (Sao Paulo). 2018 Oct 11;73(suppl 1):e565s. doi: 10.6061/clinics/2018/e565s.
Pharmacogenetics, a major component of individualized or precision medicine, relies on human genetic diversity. The remarkable developments in sequencing technologies have revealed that the number of genetic variants modulating drug action is much higher than previously thought and that a true personalized prediction of drug response requires attention to rare mutations (minor allele frequency, MAF<1%) in addition to polymorphisms (MAF>1%) in pharmacogenes. This has major implications for the conceptual development and clinical implementation of pharmacogenetics. Drugs used in cancer treatment have been major targets of pharmacogenetics studies, encompassing both germline polymorphisms and somatic variants in the tumor genome. The present overview, however, has a narrower scope and is focused on germline cancer pharmacogenetics, more specifically, on drug/gene pairs for which pharmacogenetics-informed prescription guidelines have been published by the Clinical Pharmacogenetics Implementation Consortium and/or the Dutch Pharmacogenetic Working Group, namely, thiopurines/TPMT, fluoropyrimidines/UGT1A1, irinotecan/UGT1A1 and tamoxifen/CYP2D6. I begin by reviewing the general principles of pharmacogenetics-informed prescription, pharmacogenetics testing and the perceived barriers to the adoption of routine pharmacogenetics testing in clinical practice. Then, I highlight aspects of the pharmacogenetics testing of the selected drug-gene pairs and finally present pharmacogenetics data from Brazilian studies pertinent to these drug-gene pairs. I conclude with the notion that pharmacogenetics testing has the potential to greatly benefit patients by enabling precision medicine applied to drug therapy, ensuring better efficacy and reducing the risk of adverse effects.
药物遗传学作为个体化或精准医学的一个主要组成部分,依赖于人类遗传多样性。测序技术的显著发展表明,调节药物作用的基因变异数量比以前认为的要多得多,而且对药物反应进行真正的个性化预测除了要关注药物基因中的多态性(次要等位基因频率,MAF>1%)外,还需要关注罕见突变(次要等位基因频率,MAF<1%)。这对药物遗传学的概念发展和临床应用具有重大意义。癌症治疗中使用的药物一直是药物遗传学研究的主要对象,包括肿瘤基因组中的种系多态性和体细胞变异。然而,本综述的范围较窄,专注于种系癌症药物遗传学,更具体地说,关注临床药物遗传学实施联盟和/或荷兰药物遗传学工作组已发布药物遗传学指导处方指南的药物/基因对,即硫唑嘌呤/硫嘌呤甲基转移酶(TPMT)、氟嘧啶/尿苷二磷酸葡萄糖醛酸基转移酶1A1(UGT1A1)、伊立替康/UGT1A1和他莫昔芬/细胞色素P450 2D6(CYP2D6)。我首先回顾药物遗传学指导处方、药物遗传学检测的一般原则以及临床实践中采用常规药物遗传学检测所面临的公认障碍。然后,我强调所选药物-基因对的药物遗传学检测的各个方面,最后展示巴西研究中与这些药物-基因对相关的药物遗传学数据。我的结论是,药物遗传学检测有可能通过将精准医学应用于药物治疗,确保更好的疗效并降低不良反应风险,从而使患者大大受益。