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药物诱导的尖端扭转型室性心动过速的药物遗传学方面:改善临床药物开发和处方的潜在工具。

Pharmacogenetic aspects of drug-induced torsade de pointes: potential tool for improving clinical drug development and prescribing.

作者信息

Shah Rashmi R

机构信息

Medicines and Healthcare products Regulatory Agency, London, United Kingdom.

出版信息

Drug Saf. 2004;27(3):145-72. doi: 10.2165/00002018-200427030-00001.

Abstract

Drug-induced torsade de pointes (TdP) has proved to be a significant iatro-genic cause of morbidity and mortality and a major reason for the withdrawal of a number of drugs from the market in recent times. Enzymes that metabolise many of these drugs and the potassium channels that are responsible for cardiac repolarisation display genetic polymorphisms. Anecdotal reports have suggested that in many cases of drug-induced TdP, there may be a concealed genetic defect of either these enzymes or the potassium channels, giving rise to either high plasma drug concentrations or diminished cardiac repolarisation reserve, respectively. The presence of either of these genetic defects may predispose a patient to TdP, a potentially fatal adverse reaction, even at therapeutic dosages of QT-prolonging drugs and in the absence of other risk factors. Advances in pharmacogenetics of drug metabolising enzymes and pharmacological targets, together with the prospects of rapid and inexpensive genotyping procedures, promise to individualise and improve the benefit/risk ratio of therapy with drugs that have the potential to cause TdP. The qualitative and the quantitative contributions of these genetic defects in clinical cases of TdP are unclear because not all of the patients with TdP are routinely genotyped and some relevant genetic mutations still remain to be discovered. There are regulatory guidelines that recommend strategies aimed at uncovering the risk of TdP associated with new chemical entities during their development. There are also a number of guidelines that recommend integrating pharmacogenetics in this process. This paper proposes a strategy for integrating pharmacogenetics into drug development programmes to optimise association studies correlating genetic traits and endpoints of clinical interest, namely failure of efficacy or development of repolarisation abnormalities. Until pharmacogenetics is carefully integrated into all phases of development of QT-prolonging drugs and large-scale studies are undertaken during their post-marketing use to determine the genetic components involved in induction of TdP, routine genotyping of patients remains unrealistic. Even without this pharmacogenetic data, the clinical risk of TdP can already be greatly minimised. Clinically, a substantial proportion of cases of TdP are due to the use of either high or usual dosages of drugs with potential to cause TdP in the presence of factors that inhibit drug metabolism. Therefore, choosing the lowest effective dose and identifying patients with these non-genetic risk factors are important means of minimising the risk of TdP. In view of the common secondary pharmacology shared by these drugs, a standard set of contraindications and warnings have evolved over the last decade. These include factors responsible for pharmacokinetic or pharmacodynamic drug interactions. Among the latter, the more important ones are bradycardia, electrolyte imbalance, cardiac disease and co-administration of two or more QT-prolonging drugs. In principle, if large scale prospective studies can demonstrate a substantial genetic component, pharmacogenetically driven prescribing ought to reduce the risk further. However, any potential benefits of pharmacogenetics will be squandered without any reduction in the clinical risk of TdP if physicians do not follow prescribing and monitoring recommendations.

摘要

药物性尖端扭转型室速(TdP)已被证明是发病率和死亡率的一个重要医源性原因,也是近年来许多药物撤市的主要原因。代谢这些药物中许多药物的酶以及负责心脏复极的钾通道存在基因多态性。轶事报道表明,在许多药物性TdP病例中,可能存在这些酶或钾通道的隐性遗传缺陷,分别导致血浆药物浓度升高或心脏复极储备降低。这些遗传缺陷中的任何一种都可能使患者易患TdP,这是一种潜在致命的不良反应,即使在使用延长QT药物的治疗剂量且无其他危险因素的情况下也是如此。药物代谢酶和药理学靶点的药物基因组学进展,以及快速且廉价的基因分型程序的前景,有望实现个体化并改善有可能导致TdP的药物治疗的获益/风险比。这些遗传缺陷在TdP临床病例中的定性和定量贡献尚不清楚,因为并非所有TdP患者都进行常规基因分型,而且一些相关基因突变仍有待发现。有监管指南推荐旨在在新化学实体研发过程中揭示与TdP相关风险的策略。也有一些指南建议在这个过程中整合药物基因组学。本文提出了一种将药物基因组学整合到药物研发计划中的策略,以优化关联研究,将遗传特征与临床关注的终点相关联,即疗效不佳或复极异常的发生。在将药物基因组学仔细整合到延长QT药物研发的所有阶段并在其上市后使用期间进行大规模研究以确定参与TdP诱导的遗传成分之前,对患者进行常规基因分型仍然不现实。即使没有这些药物基因组学数据,TdP的临床风险也已经可以大大降低。临床上,相当一部分TdP病例是由于在存在抑制药物代谢的因素时使用了高剂量或常规剂量的有可能导致TdP的药物。因此,选择最低有效剂量并识别具有这些非遗传危险因素的患者是降低TdP风险的重要手段。鉴于这些药物共有的常见次要药理学特性,在过去十年中已经形成了一套标准的禁忌证和警示。这些包括负责药代动力学或药效学药物相互作用的因素。在后者中,更重要的是心动过缓、电解质失衡、心脏病以及两种或更多种延长QT药物的联合使用。原则上,如果大规模前瞻性研究能够证明存在大量遗传成分,基于药物基因组学的处方应该能够进一步降低风险。然而,如果医生不遵循处方和监测建议,药物基因组学的任何潜在益处都将被浪费,而TdP的临床风险不会有任何降低。

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