Ensom M H, Chang T K, Patel P
Faculty of Pharmaceutical Sciences, The University of British Columbia, Vancouver, Canada.
Clin Pharmacokinet. 2001;40(11):783-802. doi: 10.2165/00003088-200140110-00001.
Genetic variability in drug response occurs as a result of molecular alterations at the level of drug-metabolising enzymes, drug targets/receptors, and drug transport proteins. In this paper, we discuss the possibility that therapeutic drug monitoring (TDM) in the future will involve not the mere measurement and interpretation of drug concentrations but will include both traditional TDM and pharmacogenetics-oriented TDM. In contrast to traditional TDM, which cannot be performed until after a drug is administered to the patient. pharmacogenetics-oriented TDM can be conducted even before treatment begins. Other advantages of genotyping over traditional TDM include, but are not limited to, the following: (i) it does not require the assumption of steady-state conditions (or patient compliance) for the interpretation of results; (ii) it can often be performed less invasively (with saliva, hair root or buccal swab samples); (iii) it can provide predictive value for multiple drugs [e.g. a number of cytochrome P450 (CYP) 2D6, CYP2C 19 or CYP2C9 substrates] rather than a single drug; (iv) it provides mechanistic, instead of merely descriptive, information; and (v) it is constant over an individual's lifetime (and not influenced by concurrent drug administration, alteration in hormonal levels or disease states). Pharmacogenetic information can be applied a priori for initial dose stratification and identification of cases where certain drugs are simply not effective. However, traditional TDM will still be required for all of the reasons that we use it now. In current clinical practice, pharmacogenetic testing is performed for only a few drugs (e.g. mercaptopurine, thioguanine, azathioprine, trastuzumab and tacrine) and in a limited number of teaching hospitals and specialist academic centres. We propose that other drugs (e.g. warfarin, phenytoin, codeine, oral hypoglycaemics, tricyclic antidepressants, aminoglycosides, digoxin, cyclosporin, cyclophosphamide, ifosfamide, theophylline and clozapine) are potential candidates for pharmacogenetics-oriented TDM. However, prospective studies of phaymacogenetics-oriented TDM must be performed to determine its efficacy and cost effectiveness in optimising therapeutic effects while minimising toxicity. In the future, in addition to targeting a patient's drug concentrations within a therapeutic range, pharmacists are likely to be making dosage recommendations for individual drugs on the basis of the individual patient's genotype. As we enter the era of personalised drug therapy, we will be able to identify not only the best drug to be administered to a particular patient, but also the most effective and safest dosage from the outset of therapy.
药物反应中的遗传变异性是由药物代谢酶、药物靶点/受体和药物转运蛋白水平的分子改变引起的。在本文中,我们讨论了未来治疗药物监测(TDM)可能不仅涉及药物浓度的单纯测量和解释,还将包括传统TDM和面向药物遗传学的TDM。与传统TDM不同,传统TDM在药物给予患者后才能进行,而面向药物遗传学的TDM甚至在治疗开始前就能进行。基因分型相对于传统TDM的其他优势包括但不限于以下几点:(i)解释结果时不需要稳态条件(或患者依从性)的假设;(ii)通常可以采用侵入性较小的方式进行(使用唾液、发根或口腔拭子样本);(iii)它可以为多种药物[例如一些细胞色素P450(CYP)2D6、CYP2C19或CYP2C9底物]而不是单一药物提供预测价值;(iv)它提供的是机制性信息而非仅仅是描述性信息;(v)它在个体一生中是恒定的(不受同时使用的药物、激素水平变化或疾病状态的影响)。药物遗传学信息可以先验地应用于初始剂量分层以及确定某些药物根本无效的情况。然而,出于我们现在使用传统TDM的所有原因,它仍然是必需的。在当前临床实践中,仅对少数药物(如巯嘌呤、硫鸟嘌呤、硫唑嘌呤、曲妥珠单抗和他克林)进行药物遗传学检测,且仅在少数教学医院和专科院校中心进行。我们建议其他药物(如华法林、苯妥英、可待因、口服降糖药、三环类抗抑郁药、氨基糖苷类、地高辛、环孢素、环磷酰胺、异环磷酰胺、茶碱和氯氮平)是面向药物遗传学TDM的潜在候选药物。然而,必须进行面向药物遗传学TDM的前瞻性研究,以确定其在优化治疗效果同时最小化毒性方面的疗效和成本效益。在未来,除了将患者的药物浓度控制在治疗范围内,药剂师可能会根据个体患者的基因型为个别药物制定剂量建议。随着我们进入个性化药物治疗时代,我们不仅能够确定给予特定患者的最佳药物,还能从治疗开始就确定最有效和最安全的剂量。