药物代谢动力学指导血友病A治疗的中国专家共识

Chinese expert consensus on pharmacokinetics guided treatment for hemophilia A

血友病A是一种以凝血因子Ⅷ(FactorⅧ,FⅧ)缺乏,临床表现为自发性或者损伤后过度出血的X染色体连锁隐性遗传病。血友病A目前主要治疗方式是替代性输注FⅧ,但对于输注的剂量、频次大多仍采用固定输注模式,并不能达到个体化治疗原则下的最优疗效。影响FⅧ替代治疗疗效的因素中,个体对FⅧ产品的药物代谢动力学(Pharmacokinetics,PK)差异是一个重要因素。临床对于PK指导下的个体化FⅧ替代治疗认知尚不充分,因此本文就FⅧ的PK特征、分析模型、临床应用场景与具体治疗方案制定进行分析,并为临床实践提供指导意见。

Hemophilia A is an X-chromosome-linked recessive genetic disease that lacks coagulation factorⅧ(FactorⅧ,FⅧ)and is clinically manifested as spontaneous or excessive bleeding after injury.The current main treatment for hemophilia A is alternative infusion of FⅧ,but the fixed infusion mode is still used for the dosage and frequency of infusion,which cannot achieve the optimal curative effect under the principle of individualized treatment.Among the factors that affect the efficacy of FⅧreplacement therapy,the difference in the pharmacokinetics(PK)of FⅧproducts by individuals is an important factor.The clinical understanding of individualized FⅧreplacement therapy under the guidance of PK is not sufficient.Therefore,this article reviews the PK characteristics,analysis models,clinical application scenarios and specific treatment plan formulation of FⅧ.