van Valkengoed Daan W, Krekels Elke H J, Knibbe Catherijne A J
Division of Systems Pharmacology and Pharmacy, Leiden Academic Center for Drug Research, Leiden University, Leiden, The Netherlands.
Certara Inc, Princeton, NJ, USA.
Clin Pharmacokinet. 2025 Feb;64(2):173-192. doi: 10.1007/s40262-024-01444-6. Epub 2024 Dec 7.
Scaling approaches are used to describe or predict clearance for paediatric or obese populations from normal-weight adult values. Theoretical allometry assumes the existence of a universal bodyweight-based scaling relationship. Although theoretical allometry is highly disputed, it is commonly applied in pharmacological data analyses and clinical practice. The aim of the current review is to (1) increase pharmacologists' understanding of theoretical allometry to better understand the (implicit) assumptions and (dis)advantages and (2) highlight important methodological considerations with the application of this methodology. Theoretical allometry originated in an empirical, and later debated, observation by Kleiber of a scaling exponent of 0.75 between basal metabolic rate and body mass of mammals. The mathematical framework of West, Brown, and Enquist provides one possible explanation for this value. To date, multiple key assumptions of this framework have been disputed or disproven, and an increasing body of evidence is emerging against the existence of one universal allometric exponent. The promise of ease and universality of use that comes with theoretical approaches may be the reason they are so strongly sought after and defended. However, ecologists have suggested that the theory should move from a 'Newtonian approach', in which physical explanations are sought for a universal law and variability is of minor importance, to a 'Darwinian approach', in which variability is considered of primary importance for which evolutionary explanations can be found. No scientific support was found for the application of allometry for within-species scaling, so the application of basal metabolic rate-based scaling principles to clearance scaling remains unsubstantiated. Recent insights from physiologically based modelling approaches emphasise the interplay between drugs with different properties and physiological variables that underlie drug clearance, which drives the variability in the allometric scaling exponent in the field of pharmacology. To deal with this variability, drug-specific or patient-specific adaptations to theoretical allometric scaling are proposed, that introduce empiric elements and reduce the universality of the theory. The use of allometric scaling with an exponent of 0.75 may hold empirical merit for paediatric populations, except for the youngest individuals (aged ≤ 5 years). Nevertheless, biological interpretations and extrapolation potential attributed to models based on 0.75 allometric scaling are theoretically unfounded, and merits of the empirical application of this function should, as for all models, always be supported by appropriate model validation procedures. In this respect, it is not the value of the allometric exponent but the description and prediction of individual clearance values and drug concentrations that are of primary interest.
缩放方法用于从正常体重成年人的值描述或预测儿科或肥胖人群的清除率。理论异速生长法假定存在基于体重的通用缩放关系。尽管理论异速生长法备受争议,但它在药理学数据分析和临床实践中仍被广泛应用。本综述的目的是:(1)增强药理学家对理论异速生长法的理解,以便更好地理解(隐含的)假设和(优)缺点;(2)强调应用该方法时的重要方法学考量。理论异速生长法起源于克莱伯的一项经验观察,该观察后来引发了争议,即哺乳动物的基础代谢率与体重之间的缩放指数为0.75。韦斯特、布朗和恩奎斯特的数学框架为这一数值提供了一种可能的解释。迄今为止,该框架的多个关键假设已受到质疑或被证伪,并且越来越多的证据表明不存在一个通用的异速生长指数。理论方法所带来的使用简便性和通用性的前景,可能是它们备受追捧和捍卫的原因。然而,生态学家建议,该理论应从“牛顿方法”(即寻求物理解释以得出通用定律且变异性不太重要的方法)转向“达尔文方法”(即认为变异性至关重要且可以找到进化解释的方法)。未发现有科学依据支持将异速生长法用于种内缩放,因此将基于基础代谢率的缩放原理应用于清除率缩放仍缺乏依据。基于生理的建模方法的最新见解强调了具有不同性质的药物与药物清除所依据的生理变量之间的相互作用,这推动了药理学领域异速生长缩放指数的变异性。为应对这种变异性,有人提出针对理论异速生长缩放进行药物特异性或患者特异性调整,这引入了经验元素并降低了该理论的通用性。对于儿科人群,使用指数为0.75的异速生长缩放可能具有经验价值,但最年幼的个体(年龄≤5岁)除外。然而从理论上讲,基于0.75异速生长缩放的模型所具有的生物学解释和外推潜力是没有根据的,并且与所有模型一样,该函数经验应用的优点应始终通过适当的模型验证程序来支持。在这方面,主要关注的不是异速生长指数的值,而是个体清除率值和药物浓度的描述与预测。