Chan P L, Holford N H
Division of Pharmacology and Clinical Pharmacology, School of Medicine, University of Auckland, Private Bag 92019, Auckland 1030, New Zealand.
Annu Rev Pharmacol Toxicol. 2001;41:625-59. doi: 10.1146/annurev.pharmtox.41.1.625.
Degenerative diseases are characterized by a worsening of disease status over time. The rate of deterioration is determined by the natural rate of progression of the disease and by the effect of drug treatments. A goal of drug treatment is to slow disease progression. Drug treatments can be categorized as symptomatic or protective. Symptomatic treatments do not affect the rate of disease progression whereas protective treatments have the ability to slow disease progression down. Many current methods for describing disease progression have two common drawbacks: a linear relationship between time and disease status is assumed, and within- and between-subject variability is ignored. Disease progress models combined with pharmacokinetic pharmacodynamic models and hierarchical random effects statistical models provide insights into understanding the time course and management of degenerative disease.
退行性疾病的特点是疾病状态随时间推移而恶化。恶化速度由疾病的自然进展速度和药物治疗效果决定。药物治疗的一个目标是减缓疾病进展。药物治疗可分为对症治疗或保护性治疗。对症治疗不影响疾病进展速度，而保护性治疗有能力减缓疾病进展。目前许多描述疾病进展的方法有两个共同缺点：假定时间与疾病状态之间存在线性关系，并且忽略了个体内部和个体之间的变异性。疾病进展模型与药代动力学药效学模型和分层随机效应统计模型相结合，有助于深入理解退行性疾病的病程和管理。
