基于药动学的A型血友病的凝血因子Ⅷ个体治疗的研究进展

Pharmacokinetics-Informed Individualized Dosing of FactorⅧin Patients with Haemophilia A

A型血友病是由凝血因子Ⅷ(FⅧ)缺乏而引起的出血性疾病,患者需长期补充FⅧ进行治疗。然而不同患者的治疗目标和药动学特征存在较大差异,依据个体药动学参数和需求制定个体的给药方案,能够有效地预防出血,减轻患者经济负担,具有显著的疗效优越性。近年来,基于药动学进行个体化给药得到快速发展,国内外已有基于群体药动学和最大后验贝叶斯法的给药方案设计工具,协助医务工作者实现个体化给药。笔者将阐述FⅧ产品的药动学特征、影响因素以及基于药动学指导的个体化治疗方法和临床应用,以期为A型血友病患者的个体化给药研究及实践提供参考。

Haemophilia A is a clotting disorder caused by factorⅧdeficiency.Patients who have haemophilia A need long-term factorⅧreplacement.However,different patients have different goals of treatment and pharmacokinetics characteristics.Developing individualized dosing based on individual patients'pharmacokinetics and goals has shown its advantage in effectively preventing bleeding and reducing the economic burden.In recent years,there is a growing use of pharmacokinetics-informed individualized dosing in clinical practice.To further assist the calculation of individualized dosing,researchers have developed dosing tools based on population pharmacokinetics and maximum a posteriori Bayesian(MAPB)estimation in China and other countries.In this review,we summarized the pharmacokinetics characteristics of factorⅧand its influential factors.Moreover,the individualized dosing methods of factorⅧbased on pharmacokinetics,especially with MAPB approach are described to optimize the clinical management of patients with Haemophilia A.